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Hemorrhagic Disorders HELP
Based on 37,428 articles published since 2008
|||| 14 

These are the 37428 published articles about Hemorrhagic Disorders that originated from Worldwide during 2008-2019.
 
+ Citations + Abstracts
Pages: 1 · 2 · 3 · 4 · 5 · 6 · 7 · 8 · 9 · 10 · 11 · 12 · 13 · 14 · 15 · 16 · 17 · 18 · 19 · 20
1 Guideline Consensus Statement of the Indian Academy of Pediatrics in Diagnosis and Management of Hemophilia. 2018

Sachdeva, Anupam / Gunasekaran, Vinod / Ramya, H N / Dass, Jasmita / Kotwal, Jyoti / Seth, Tulika / Das, Satyaranjan / Garg, Kapil / Kalra, Manas / Sirisha, Rani S / Prakash, Anand / Anonymous1860959. ·Sir Ganga Ram Hospital, New Delhi, India. Correspondence to: Dr. Anupam Sachdeva, Director, Pediatric Hematology Oncology and Bone Marrow Transplantation unit, Institute of Child Health, Sir Ganga Ram Hospital, New Delhi 110 060, India. anupamace@yahoo.co.in. · Sir Ganga Ram Hospital, New Delhi, India. · All India Institute of Medical Sciences, New Delhi, India. · Army Hospital Research and Referral, New Delhi, India. · Sawai Man Singh Medical College, Jaipur, India. · Indraprastha Apollo Hospitals, New Delhi, India. · Rainbow Children's Hospital, Hyderabad, India. · St Johns Medical College Hospital, Bangalore, India. ·Indian Pediatr · Pubmed #30129541.

ABSTRACT: JUSTIFICATION: Despite having standard principles of management of hemophilia, treatment differs in various countries depending on available resources. Guideline for management of hemophilia in Indian setting is essential. PROCESS: Indian Academy of Pediatrics conducted a consultative meeting on Hemophilia on 18th September, 2016 in New Delhi, which was attended by experts in the field working across India. Scientific literature was reviewed, and guidelines were drafted. All expert committee members reviewed the final manuscript. OBJECTIVE: To bring out consensus guidelines in diagnosis and management of Hemophilia in India. RECOMMENDATIONS: Specific factor assays confirm diagnosis and classify hemophilia according to residual factor activity (mild 5-40%, moderate 1-5%, severe <1%). Genetic testing helps in identifying carriers, and providing genetic counseling and prenatal diagnosis. Patients with hemophilia should be managed by multi-specialty team approach. Continuous primary prophylaxis (at least low-dose regimen of 10-20 IU/kg twice or thrice per week) is recommended in severe hemophilia with dose tailored as per response. Factor replacement remains the mainstay of treating acute bleeds (dose and duration depends on body weight, site and severity of bleed). Factor concentrates (plasma derived or recombinant), if available, are preferred over blood components. Other supportive measures (rest, ice, compression, and elevation) should be instantly initiated. Long-term complications include musculoskeletal problems, development of inhibitors and transfusion-transmitted infections, which need monitoring. Adequate vaccination of children with hemophilia (with precautions) is emphasized.

2 Guideline Radiation Therapy for Solitary Plasmacytoma and Multiple Myeloma: Guidelines From the International Lymphoma Radiation Oncology Group. 2018

Tsang, Richard W / Campbell, Belinda A / Goda, Jayant S / Kelsey, Chris R / Kirova, Youlia M / Parikh, Rahul R / Ng, Andrea K / Ricardi, Umberto / Suh, Chang-Ok / Mauch, Peter M / Specht, Lena / Yahalom, Joachim. ·Department of Radiation Oncology, Princess Margaret Cancer Centre, University of Toronto, Toronto, Ontario, Canada. Electronic address: richard.tsang@rmp.uhn.on.ca. · Division of Radiation Oncology, Peter MacCallum Cancer Centre, Melbourne, Victoria, Australia. · Department of Radiation Oncology, Tata Memorial Centre, Mumbai, India. · Department of Radiation Oncology, Duke University Medical Center, Durham, North Carolina. · Department of Radiation Therapy, Institut Curie, Paris, France. · Department of Radiation Oncology, Rutgers Cancer Institute of New Jersey, New Brunswick, New Jersey. · Department of Radiation Oncology, Brigham and Women's Hospital and Dana Farber Cancer Institute, Boston, Massachusetts. · Radiation Oncology Unit, Department of Oncology, University of Torino, Torino, Italy. · Department of Radiation Oncology, Yonsei University College of Medicine, Seoul, South Korea. · Department of Oncology, Rigshospitalet, University of Copenhagen, Copenhagen, Denmark. · Department of Radiation Oncology, Memorial Sloan-Kettering Cancer Center, New York, New York. ·Int J Radiat Oncol Biol Phys · Pubmed #29976492.

ABSTRACT: PURPOSE: To develop guidelines for the work-up and radiation therapy (RT) management of patients with plasma cell neoplasms. METHODS AND MATERIALS: A literature review was conducted covering staging, work-up, and RT management of plasma cell neoplasms. Guidelines were developed through consensus by an international panel of radiation oncologists with expertise in these diseases, from the International Lymphoma Radiation Oncology Group. RT volume definitions are based on the International Commission on Radiation Units and Measurements. RESULTS: Plasma cell neoplasms account for approximately one-fifth of mature B-cell neoplasms in the United States. The majority (∼95%) are diagnosed as multiple myeloma, in which there has been tremendous progress in systemic therapy approaches with novel drugs over the last 2 decades, resulting in improvements in disease control and survival. In contrast, a small proportion of patients with plasma cell neoplasms present with a localized plasmacytoma in the bone, or in extramedullary (extraosseous) soft tissues, and definitive RT is the standard treatment. RT provides long-term local control in the solitary bone plasmacytomas and is potentially curative in the extramedullary cases. This guideline reviews the diagnostic work-up, principles, and indications for RT, target volume definition, treatment planning, and follow-up procedures for solitary plasmacytoma. Specifically, detailed recommendations for RT volumes and dose/fractionation are provided, illustrated with specific case scenarios. The role of palliative RT in multiple myeloma is also discussed. CONCLUSIONS: The International Lymphoma Radiation Oncology Group presents a standardized approach to the use and implementation of definitive RT in solitary plasmacytomas. The modern principles outlining the supportive role of palliative RT in multiple myeloma in an era of novel systemic therapies are also discussed.

3 Guideline BSH Guideline: management of thrombotic and haemostatic issues in paediatric malignancy. 2018

Sibson, Keith R / Biss, Tina T / Furness, Caroline L / Grainger, John D / Hough, Rachael E / Macartney, Christine / Payne, Jeanette H / Chalmers, Elizabeth A / Anonymous610935. ·Department of Haematology, Great Ormond Street Hospital, London, UK. · The Newcastle Upon Tyne Hospitals NHS Foundation Trust, Newcastle Upon Tyne, UK. · Bristol Haematology Oncology Centre, Bristol, UK. · Royal Manchester Children's Hospital, Manchester, UK. · University College Hospital, London, UK. · Royal Belfast Children's Hospital, Belfast, UK. · Sheffield Children's Hospital, Sheffield, UK. · Royal Hospital for Children, Glasgow, UK. ·Br J Haematol · Pubmed #29384193.

ABSTRACT: -- No abstract --

4 Guideline [Preventative and therapeutic relapse strategies after allogeneic hematopoietic stem cell transplantation: Guidelines from the Francophone society of bone marrow transplantation and cellular therapy (SFGM-TC)]. 2017

Yafour, Nabil / Beckerich, Florence / Bulabois, Claude Eric / Chevallier, Patrice / Daguindau, Étienne / Dumesnil, Cécile / Guillaume, Thierry / Huynh, Anne / Levrat, Stavroula Masouridi / Menard, Anne-Lise / Michallet, Mauricette / Pautas, Cécile / Poiré, Xavier / Ravinet, Aurelie / Yakoub-Agha, Ibrahim / Bazarbachi, Ali. ·Établissement hospitalier et universitaire 1(er) Novembre 1954, service d'hématologie et de thérapie cellulaire, BP 4166, 31000 Ibn Rochd, Oran, Algérie; Université d'Oran 1, Ahmed Ben Bella, faculté de médecine, Oran, Algérie. · Hôpital Henri-Mondor, service d'hématologie et de thérapie cellulaire, boulevard Maréchal-de-Lattre-de-Tassigny, 94000 Créteil, France. · Centre hospitalier universitaire, service d'hématologie clinique, 38043 Grenoble cedex 9, France. · CHU de Nantes, service d'hématologie clinique, Hôtel-Dieu, place Alexis-Ricordeau, 44035 Nantes, France. · CHRU de Besançon, service d'hématologie, 3, boulevard Fleming, 25000 Besancon, France. · CHU de Rouen, service d'hémato-oncologie pédiatrique, 1, rue de Germont, 76031 Rouen cedex, France. · IUCT-Oncopole, service d'hématologie et de greffe de cellules souches hématopoïétiques, 1, rue Irène-Joliot-Curie, 31059 Toulouse cedex, France. · Division of hematology, department of medical specialties, Geneva university hospitals, 4, rue Gabrielle-Perret-Gentil, 1205 Geneva, Suisse. · Centre Henri-Becquerel, service d'hématologie clinique, rue d'Amiens, CS 11516, 76038 Rouen cedex 1, France. · Centre hospitalier Lyon Sud, service d'hématologie clinique, 165, chemin du Grand-Revoyet, 69495 Pierre-Bénite cedex, France. · Universitaires Saint-Luc, section of hematology cliniques, 1200 Brussels, Belgique. · CHU Clermont-Ferrand, service de thérapie cellulaire et d'hématologie clinique adulte, 63100 Clermont-Ferrand, France; Université Clermont-Auvergne, EA7453 et CIC-501, 63003 Clermont-Ferrand, France. · CHRU de Lille, université de Lille 2, département de maladie du sang, LIRIC Inserm U995, 59000 Lille, France. Electronic address: ibrahim.yakoubagha@chru-lille.fr. · American university of Beirut, medical center, P.O. Box 113-6044 Beirut, Liban. ·Bull Cancer · Pubmed #29179894.

ABSTRACT: Disease relapse remains the first cause of mortality of hematological malignancies after allogeneic hematopoietic stem cell transplantation (allo-HCT). The risk of recurrence is elevated in patients with high-risk cytogenetic or molecular abnormalities, as well as when allo-HCT is performed in patients with refractory disease or with persistent molecular or radiological (PET-CT scan) residual disease. Within the frame of the 7th annual workshops of the francophone society for bone marrow transplantation and cellular therapy, the working group reviewed the literature in order to elaborate unified guidelines for the prevention and treatment of relapse after allo-HCT. For high risk AML and MDS, a post transplant maintenance strategy is possible, using hypomethylating agents or TKI anti-FLT3 when the target is present. For Philadelphia positive ALL, there was a consensus for the use of post-transplant TKI maintenance. For lymphomas, there are no strong data on the use of post-transplant maintenance, and hence a preemptive strategy is recommended based on modulation of immunosuppression, close follow-up of donor chimerism, and donor lymphocytes infusion. For multiple myeloma, even though the indication of allo-HCT is controversial, our recommendation is post transplant maintenance using bortezomib, due to its a good toxicity profile without increasing the risk of GVHD.

5 Guideline [Allogeneic haematopoietic cell transplantation for indolent lymphomas: Guidelines from the Francophone Society Bone Marrow Transplantation and Cellular Therapy (SFGM-TC)]. 2017

Gauthier, Jordan / Chantepie, Sylvain / Bouabdallah, Krimo / Jost, Edgar / Nguyen, Stéphanie / Gac, Anne-Claire / Damaj, Gandhi / Duléry, Rémy / Michallet, Mauricette / Delage, Jérémy / Lewalle, Philippe / Morschhauser, Franck / Salles, Gilles / Yakoub-Agha, Ibrahim / Cornillon, Jérôme. ·CHRU de Lille, pôle spécialités médicales et gérontologie, service des maladies du sang, secteur allogreffe de cellules souches hématopoïétiques, 59037 Lille, France; Université de Lille, UFR médecine, 59000 Lille, France. · AP-HP, hôpital La Pitié-Salpêtrière, service d'hématologie, 75013 Paris, France. · CHU de Caen, service d'hématologie, 14033 Caen, France. · Universitätsklinikum Aachen, Klinik für Onkologie, Hämatologie und Stammzelltransplantation, Aachen, Allemagne. · CHU Haut-Lévêque, service d'hématologie, 33600 Pessac, France. · AP-HP, hôpital Saint-Antoine, service d'hématologie, 75012 Paris, France. · CHU de Lyon, service d'hématologie, 69310 Pierre-Bénite, France. · CHU de Montpellier, service d'hématologie, 34295 Montpellier, France. · Université libre de Bruxelles, institut Jules-Bordet, service d'hématologie, Bruxelles, Belgique. · CHRU de Lille, pôle spécialités médicales et gérontologie, service des maladies du sang, secteur allogreffe de cellules souches hématopoïétiques, 59037 Lille, France; CHU de Lille, université de Lille2, LIRIC Inserm U995, 59000 Lille, France. · Institut de cancérologie Lucien-Neuwirth, département d'hématologie clinique, 42271 Saint-Priest-en-Jarez, France. Electronic address: jerome.cornillon@icloire.fr. ·Bull Cancer · Pubmed #29173973.

ABSTRACT: Despite great improvements in the outcome of patients with lymphoma, some may still relapse or present with primary refractory disease. In these situations, allogeneic hematopoietic cell transplantation is a potentially curative option, this is true particularly the case of relapse after autologous stem cell transplantation. Recently, novel agents such as anti-PD1 and BTK inhibitors have started to challenge the use of allogeneic hematopoietic cell transplantation for relapsed or refractory lymphoma. During the 2016 annual workshop of the Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC), we performed a comprehensive review of the literature published in the last 10 years and established guidelines to clarify the indications and transplant modalities in this setting. This paper specifically reports on our conclusions regarding indolent lymphomas, mainly follicular lymphoma and chronic lymphocytic leukemia.

6 Guideline Estimating and interpreting the pharmacokinetic profiles of individual patients with hemophilia A or B using a population pharmacokinetic approach: communication from the SSC of the ISTH. 2017

Iorio, A / Blanchette, V / Blatny, J / Collins, P / Fischer, K / Neufeld, E. ·Department of Health Research, Methods, Evidence and Impact, McMaster University, Hamilton, Ontario, Canada. · Department of Medicine, McMaster University, Hamilton, Ontario, Canada. · Division of Hematology/Oncology, Hospital for Sick Children and Department of Pediatrics, University of Toronto, Toronto, Ontario, Canada. · Department of Pediatric Hematology, University Hospital Brno, Brno, Czech Republic. · Arthur Bloom Haemophilia Centre, School of Medicine, University Hospital of Wales, Cardiff University, Cardiff, UK. · Van Creveldkliniek, University Medical Center, Utrecht, the Netherlands. · St Jude Children's Research Hospital, Memphis, TN, USA. ·J Thromb Haemost · Pubmed #29119666.

ABSTRACT: -- No abstract --

7 Guideline Guidelines for the use of imaging in the management of patients with myeloma. 2017

Chantry, Andrew / Kazmi, Majid / Barrington, Sally / Goh, Vicky / Mulholland, Nicola / Streetly, Matthew / Lai, Maggie / Pratt, Guy / Anonymous360912. ·Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK. · Department of Haematology, Guys and St Thomas's NHS Foundation Trust, London, UK. · Division of Imaging Sciences & Biomedical Engineering, King's College London Department of Cancer Imaging, London, UK. · The PET Centre, Guy's and St Thomas' NHS Foundation Trust, London, UK. · Department of Radiology, Guy's and St Thomas' NHS Foundation Trust, London, UK. · Department of Radiology, Kings College Hospital NHS Foundation Trust, London, UK. · Myeloma UK, Edinburgh, UK. · Department of Haematology, University Hospital Birmingham NHS Foundation Trust, Birmingham, UK. ·Br J Haematol · Pubmed #28677897.

ABSTRACT: The role of imaging in myeloma has gained increasing importance over the past few years. The recently revised definition of myeloma from the International Myeloma Working Group (IMWG) includes cross sectional imaging as a method to define bone disease and also incorporates its use in the disease definition for patients with suspected smouldering myeloma. The National Institute for Health and Care Excellence myeloma guidelines also recommend cross sectional imaging for patients with suspected myeloma. There is also increasing use of imaging in disease assessments and the International Myeloma Working Group has recently incorporated imaging in defining new response categories of minimal residual disease negativity, with or without imaging-based evidence of disease. Plain X-rays have previously been the standard imaging modality included in a myeloma work up at presentation but evidence is mounting for use of cross-sectional modalities such as computed tomography (CT), magnetic resonance imaging (MRI) and

8 Guideline Proposal for standardized preanalytical and analytical conditions for measuring thrombin generation in hemophilia: communication from the SSC of the ISTH. 2017

Dargaud, Y / Wolberg, A S / Gray, E / Negrier, C / Hemker, H C / Anonymous2721006. ·Hospices Civils de Lyon, Clinical Haemostasis Unit, Hopital Cardiologique Louis Pradel - Universite Lyon 1, Lyon, France. · Department of Pathology and Laboratory Medicine, UNC, Chapel Hill, NC, USA. · Haemostasis Section, Biotherapeutics, National Institute for Biological Standards and Control, Potters Bar, Hertfordshire, UK. · Cardiovascular Research Institute Maastricht (CARIM), University of Maastricht, Maastricht, the Netherlands. ·J Thromb Haemost · Pubmed #28656617.

ABSTRACT: -- No abstract --

9 Guideline Multiple myeloma: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. 2017

Moreau, P / San Miguel, J / Sonneveld, P / Mateos, M V / Zamagni, E / Avet-Loiseau, H / Hajek, R / Dimopoulos, M A / Ludwig, H / Einsele, H / Zweegman, S / Facon, T / Cavo, M / Terpos, E / Goldschmidt, H / Attal, M / Buske, C / Anonymous7570904. ·Haematology Department, University Hospital Hôtel-Dieu, Nantes, France. · Clinica Universidad de Navarra, CIMA, IDISNA, Pamplona, Spain. · Erasmus Medical Center Institute, Rotterdam, The Netherlands. · University Hospital of Salamanca, IBSAL, Salamanca, Spain. · Seragnoli Institute of Hematology, School of Medicine, Bologna University, Bologna, Italy. · The Cancer Research Center of Toulouse, CRCT, INSERM U 1037, Toulouse, France. · Faculty of Medicine, University Hospital Ostrava, Ostrava, Czech Republic. · Department of Clinical Therapeutics, School of Medicine, University of Athens, Athens, Greece. · Wilhelminen Cancer Research Institute, Wilhelminenspital, Vienna, Austria. · Department of Internal Medicine II, University Hospital Wuerzburg, Wuerzburg, Germany. · Department of Hematology, VU University Medical Center, Amsterdam, The Netherlands. · University Hospital Huriez, Lille, France. · Department of Medicine, University of Heidelberg, Heidelberg. · Comprehensive Cancer Center Ulm and Department of Internal Medicine III, Institute of Experimental Cancer Research, University Hospital, Ulm, Germany. ·Ann Oncol · Pubmed #28453614.

ABSTRACT: -- No abstract --

10 Guideline Investigation and management of IgM and Waldenström-associated peripheral neuropathies: recommendations from the IWWM-8 consensus panel. 2017

D'Sa, Shirley / Kersten, Marie José / Castillo, Jorge J / Dimopoulos, Meletios / Kastritis, Efstathios / Laane, Edward / Leblond, Véronique / Merlini, Giampaolo / Treon, Steven P / Vos, Josephine M / Lunn, Michael P. ·Waldenström Clinic, Cancer Division, University College London Hospitals NHS Foundation Trust, London, UK. · Department of Haematology, Academic Medical Centre, Amsterdam, the Netherlands. · Bing Center for Waldenström Macroglobulinemia, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA, USA. · Department of Clinical Therapeutics, National and Kapodistrian University of Athens, Athens, Greece. · Department of Haematology, North Estonia Medical Centre, Tallinn, Estonia. · AP-HP Hôpital Pitié Salpêtrière, UPMC Univ. Paris 6 GRC-11, Grechy, Paris, France. · Centre for Research and Treatment of Systemic Amyloidosis, University of Pavia, Pavia, Italy. · Cancer Centre, Sint Antonius Ziekenhuis, Nieuwegein, the Netherlands. · Centre for Neuromuscular Diseases, National Hospital for Neurology and Neurosurgery, London, UK. ·Br J Haematol · Pubmed #28198999.

ABSTRACT: Paraproteinaemic neuropathies are a heterogeneous group of disorders most frequently associated with IgM monoclonal gammopathies including Waldenström macroglobulinaemia (WM). Their consequences are significant for affected patients, and their management challenging for their physicians. The variability in clinical presentation and time course hamper classification and management. The indications for invasive investigations such as cerebrospinal fluid analysis, nerve conduction tests and sensory nerve biopsies are unclear, and the optimum way to measure clinical response to treatment unknown. When to intervene and and how to treat, also present challenges to physicians. As part of its latest deliberations at the International Workshops on WM (IWWM) in London, UK (August 2014), the IWWM8 panel have proposed a consensus approach to the diagnosis and management of peripheral neuropathies associated with IgM monoclonal gammopathies, including WM. Importantly, a consensus regarding the use of clinical outcome measures and recommended models of care for this group of patients is discussed, as well as appropriate treatment interventions.

11 Guideline Multiple Myeloma, Version 3.2017, NCCN Clinical Practice Guidelines in Oncology. 2017

Kumar, Shaji K / Callander, Natalie S / Alsina, Melissa / Atanackovic, Djordje / Biermann, J Sybil / Chandler, Jason C / Costello, Caitlin / Faiman, Matthew / Fung, Henry C / Gasparetto, Cristina / Godby, Kelly / Hofmeister, Craig / Holmberg, Leona / Holstein, Sarah / Huff, Carol Ann / Kassim, Adetola / Liedtke, Michaela / Martin, Thomas / Omel, James / Raje, Noopur / Reu, Frederic J / Singhal, Seema / Somlo, George / Stockerl-Goldstein, Keith / Treon, Steven P / Weber, Donna / Yahalom, Joachim / Shead, Dorothy A / Kumar, Rashmi. · ·J Natl Compr Canc Netw · Pubmed #28188192.

ABSTRACT: Multiple myeloma (MM) is caused by the neoplastic proliferation of plasma cells. These neoplastic plasma cells proliferate and produce monoclonal immunoglobulin in the bone marrow causing skeletal damage, a hallmark of multiple myeloma. Other MM-related complications include hypercalcemia, renal insufficiency, anemia, and infections. The NCCN Multiple Myeloma Panel members have developed guidelines for the management of patients with various plasma cell dyscrasias, including solitary plasmacytoma, smoldering myeloma, multiple myeloma, systemic light chain amyloidosis, and Waldenström's macroglobulinemia. The recommendations specific to the diagnosis and treatment of patients with newly diagnosed MM are discussed in this article.

12 Guideline Guidelines for screening and management of late and long-term consequences of myeloma and its treatment. 2017

Snowden, John A / Greenfield, Diana M / Bird, Jennifer M / Boland, Elaine / Bowcock, Stella / Fisher, Abigail / Low, Eric / Morris, Monica / Yong, Kwee / Pratt, Guy / Anonymous1840894. ·Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK. · Department of Oncology and Metabolism, University of Sheffield, Sheffield, UK. · Department of Oncology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK. · University Hospitals Bristol NHS Foundation Trust, Bristol, UK. · Palliative Medicine, Queen's Centre for Oncology and Haematology, Hull and East Yorkshire Hospitals NHS Trust, Hull, UK. · King's College Hospital NHS Foundation Trust, London, UK. · University College London, London, UK. · Myeloma UK, Edinburgh, UK. · University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK. ·Br J Haematol · Pubmed #28107574.

ABSTRACT: A growing population of long-term survivors of myeloma is now accumulating the 'late effects' not only of myeloma itself, but also of several lines of treatment given throughout the course of the disease. It is thus important to recognise the cumulative burden of the disease and treatment-related toxicity in both the stable and active phases of myeloma, some of which is unlikely to be detected by routine monitoring. We summarise here the evidence for the key late effects in long-term survivors of myeloma, including physical and psychosocial consequences (in Parts 1 and 2 respectively), and recommend the use of late-effects screening protocols in detection and intervention. The early recognition of late effects and effective management strategies should lead to an improvement in the management of myeloma patients, although evidence in this area is currently limited and further research is warranted.

13 Guideline Diagnosis and Management of Waldenström Macroglobulinemia: Mayo Stratification of Macroglobulinemia and Risk-Adapted Therapy (mSMART) Guidelines 2016. 2017

Kapoor, Prashant / Ansell, Stephen M / Fonseca, Rafael / Chanan-Khan, Asher / Kyle, Robert A / Kumar, Shaji K / Mikhael, Joseph R / Witzig, Thomas E / Mauermann, Michelle / Dispenzieri, Angela / Ailawadhi, Sikander / Stewart, A Keith / Lacy, Martha Q / Thompson, Carrie A / Buadi, Francis K / Dingli, David / Morice, William G / Go, Ronald S / Jevremovic, Dragan / Sher, Taimur / King, Rebecca L / Braggio, Esteban / Novak, Ann / Roy, Vivek / Ketterling, Rhett P / Greipp, Patricia T / Grogan, Martha / Micallef, Ivana N / Bergsagel, P Leif / Colgan, Joseph P / Leung, Nelson / Gonsalves, Wilson I / Lin, Yi / Inwards, David J / Hayman, Suzanne R / Nowakowski, Grzegorz S / Johnston, Patrick B / Russell, Steven J / Markovic, Svetomir N / Zeldenrust, Steven R / Hwa, Yi L / Lust, John A / Porrata, Luis F / Habermann, Thomas M / Rajkumar, S Vincent / Gertz, Morie A / Reeder, Craig B. ·Division of Hematology, Mayo Clinic, Rochester, Minnesota. · Division of Hematology/Oncology, Mayo Clinic, Scottsdale, Arizona. · Division of Hematology/Oncology, Mayo Clinic, Jacksonville, Florida. · Division of Neurology, Mayo Clinic, Rochester, Minnesota. · Division of Hematopathology, Mayo Clinic, Rochester, Minnesota. · Division of Laboratory Genetics, Mayo Clinic, Rochester, Minnesota. · Department of Cardiovascular Diseases, Mayo Clinic, Rochester, Minnesota. · Division of Nephrology, Mayo Clinic, Rochester, Minnesota. ·JAMA Oncol · Pubmed #28056114.

ABSTRACT: Importance: Waldenström macroglobulinemia (WM), an IgM-associated lymphoplasmacytic lymphoma, has witnessed several practice-altering advances in recent years. With availability of a wider array of therapies, the management strategies have become increasingly complex. Our multidisciplinary team appraised studies published or presented up to December 2015 to provide consensus recommendations for a risk-adapted approach to WM, using a grading system. Observations: Waldenström macroglobulinemia remains a rare, incurable cancer, with a heterogeneous disease course. The major classes of effective agents in WM include monoclonal antibodies, alkylating agents, purine analogs, proteasome inhibitors, immunomodulatory drugs, and mammalian target of rapamycin inhibitors. However, the highest-quality evidence from rigorously conducted randomized clinical trials remains scant. Conclusions and Relevance: Recognizing the paucity of data, we advocate participation in clinical trials, if available, at every stage of WM. Specific indications exist for initiation of therapy. Outside clinical trials, based on the synthesis of available evidence, we recommend bendamustine-rituximab as primary therapy for bulky disease, profound hematologic compromise, or constitutional symptoms attributable to WM. Dexamethasone-rituximab-cyclophosphamide is an alternative, particularly for nonbulky WM. Routine rituximab maintenance should be avoided. Plasma exchange should be promptly initiated before cytoreduction for hyperviscosity-related symptoms. Stem cell harvest for future use may be considered in first remission for patients 70 years or younger who are potential candidates for autologous stem cell transplantation. At relapse, retreatment with the original therapy is reasonable in patients with prior durable responses (time to next therapy ≥3 years) and good tolerability to previous regimen. Ibrutinib is efficacious in patients with relapsed or refractory disease harboring MYD88 L265P mutation. In the absence of neuropathy, a bortezomib-rituximab-based option is reasonable for relapsed or refractory disease. In select patients with chemosensitive disease, autologous stem cell transplantation should be considered at first or second relapse. Everolimus and purine analogs are suitable options for refractory or multiply relapsed WM. Our recommendations are periodically updated as new, clinically relevant information emerges.

14 Guideline IgA vasculitis (Henoch-Schönlein): Case definition andguidelines for data collection, analysis, and presentation of immunisation safety data. 2017

Woerner, Andreas / Rudin, Christoph / Bonetto, Caterina / Santuccio, Carmela / Ozen, Seza / Wise, Robert P / Chandler, Rebecca / Bonhoeffer, Jan / Anonymous750892. ·Pediatric Rheumatology, University of Basel, University Children's Hospital, Basel, Switzerland. Electronic address: contact@brightoncollaboration.org. · Pediatric Nephrology, University of Basel, University Children's Hospital, Basel, Switzerland. · Italian Medicines Agency (AIFA), Rome, Italy. · Department of Pediatrics, Hacettepe University, Faculty of Medicine, Ankara, Turkey. · AstraZeneca, UK. · Uppsala Monitoring Centre, Uppsala, Sweden. · Brighton Collaboration Foundation, Switzerland; Pediatric Infectious Diseases and Vaccines, University of Basel, University Children's Hospital, Basel, Switzerland. ·Vaccine · Pubmed #28034474.

ABSTRACT: -- No abstract --

15 Guideline Guidelines of the French Society of Otorhinolaryngology (SFORL) (short version). Specific treatment of epistaxis in Rendu-Osler-Weber disease. 2017

Robard, L / Michel, J / Prulière Escabasse, V / Bequignon, E / Vérillaud, B / Malard, O / Crampette, L / Anonymous7220890. ·Service d'ORL, CHU de Caen, Avenue de la Côte-de-Nacre, 14003 Caen, France. Electronic address: robard-l@chu-caen.fr. · Service ORL, Hôpital de la Conception, CHU de Marseille, 147, Boulevard Baille, 13005 Marseille, France. · Service ORL, CHIC de Créteil, 40, Avenue de Verdun, 94000 Créteil, France. · Service ORL, Hôpital Lariboisière, AP-HP, Université Paris 7, 2, Rue Ambroise-Paré, 75475 Paris Cedex 10, France. · Service ORL, CHU de Nantes, 44000 Nantes, France. · Service ORL, CHU de Montpellier, 191, Avenue du Doyen Gaston Giraud, 34295 Montpellier cedex 5, France. ·Eur Ann Otorhinolaryngol Head Neck Dis · Pubmed #27986467.

ABSTRACT: OBJECTIVES: The authors present the guidelines of the French Oto-Rhino-Laryngology - Head and Neck Surgery Society (Société Française d'Oto-Rhino-Laryngologie et de Chirurgie de la Face et du Cou: SFORL) concerning specific treatment of epistaxis in Rendu-Osler-Weber disease. METHODS: A multidisciplinary work-group was entrusted with a review of the scientific literature on the above topic. Guidelines were drawn up, based on the articles retrieved and the group members' individual experience. They were then read over by an editorial group independent of the work group. The final version was established in a coordination meeting. The guidelines were graded as A, B, C or expert opinion, by decreasing level of evidence. RESULTS: Rendu-Osler-Weber disease is diagnosed from the presence of at least three of Curaçao's four criteria. In acute epistaxis, bidigital compression is recommended. Embolization is reserved for resistant epistaxis. Non-resorbable nasal packing and cauterization are contraindicated. Patient education is essential. Telangiectasia of the nasal mucosa can be treated by various local means. In the event of insufficient control, systemic administration of tranexamic acid is recommended.

16 Guideline Guideline for the diagnosis, treatment and response criteria for Bing-Neel syndrome. 2017

Minnema, Monique C / Kimby, Eva / D'Sa, Shirley / Fornecker, Luc-Matthieu / Poulain, Stéphanie / Snijders, Tom J / Kastritis, Efstathios / Kremer, Stéphane / Fitsiori, Aikaterini / Simon, Laurence / Davi, Frédéric / Lunn, Michael / Castillo, Jorge J / Patterson, Christopher J / Le Garff-Tavernier, Magali / Costopoulos, Myrto / Leblond, Véronique / Kersten, Marie-José / Dimopoulos, Meletios A / Treon, Steven P. ·Department of Hematology, UMC Utrecht Cancer Center, the Netherlands m.c.minnema@umcutrecht.nl. · Hematology Center, Department of Medicine, Karolinska Institutet, Stockholm, Sweden. · Cancer Division, University College London Hospitals NHS Foundation Trust, UK. · Department of Oncology and Hematology, Hôpital Universitaires de Strasbourg and Université de Strasbourg, France. · Service d'Hématologie-Immunologie-Cytogénétique, Centre Hospitalier de Valenciennes/Laboratoire d'Hématologie, Centre de Biologie et Pathologie, CHRU de Lille/INSERM, France. · Department of Neurology & Neurosurgery, Brain Center Rudolf Magnus, UMC Utrecht, The Netherlands. · Department of Clinical Therapeutics, National and Kapodistrian University of Athens, Greece. · Pôle d'Imagerie-Neuroradiologie, Hôpital de Hautepierre/CHU Strasbourg, France. · Laboratory of Hematology, Hôpital Pitié Salpêtrière, Paris, France. · Centre for Neuromuscular Disease, National Hospital for Neurology and Neurosurgery, Queen Square, London, UK. · Bing Center for Waldenstrom's Macroglobulinemia, Dana Farber Cancer Institute and Harvard Medical; School, Boston, MA, USA. · AP-HP Hôpital Pitié Salpêtrière, UPMC univ Paris, France. · Department of Hematology, Academic Medical Center, Amsterdam, the Netherlands. ·Haematologica · Pubmed #27758817.

ABSTRACT: Bing Neel syndrome is a rare disease manifestation of Waldenström's macroglobulinemia that results from infiltration of the central nervous system by malignant lymphoplasmacytic cells. In this guideline we describe the clinical symptoms, as well as the appropriate laboratory and radiological studies, that can aid in the diagnosis. The presentation of Bing Neel syndrome may be very diverse, and includes headaches, cognitive deficits, paresis, and psychiatric symptoms. The syndrome can present in patients with known Waldenström's macroglobulinemia, even in the absence of systemic progression, but also in previously undiagnosed patients. Diagnostic work-up should include cerebral spinal fluid analysis with multiparameter flow cytometry to establish B-cell clonality, protein electrophoresis and immunofixation for the detection and classification of a monoclonal protein as well as molecular diagnostic testing for immunoglobulin gene rearrangement and mutated MYD88. MRI of the brain and spinal cord is also essential. The second challenge is to expand our knowledge of prognosis and treatment outcome. Prospective clinical trials on Bing Neel syndrome patients that employ uniform treatment along with appropriate laboratory cerebral spinal fluid assessments and standardized MRI protocols will be invaluable, constituting a significant step forward in delineating treatment outcome for this intriguing disease manifestation.

17 Guideline Myeloproliferative Neoplasms, Version 2.2017, NCCN Clinical Practice Guidelines in Oncology. 2016

Mesa, Ruben / Jamieson, Catriona / Bhatia, Ravi / Deininger, Michael W / Gerds, Aaron T / Gojo, Ivana / Gotlib, Jason / Gundabolu, Krishna / Hobbs, Gabriela / Klisovic, Rebecca B / Kropf, Patricia / Mohan, Sanjay R / Oh, Stephen / Padron, Eric / Podoltsev, Nikolai / Pollyea, Daniel A / Rampal, Raajit / Rein, Lindsay A M / Scott, Bart / Snyder, David S / Stein, Brady L / Verstovsek, Srdan / Wadleigh, Martha / Wang, Eunice S / Bergman, Mary Anne / Gregory, Kristina M / Sundar, Hema. · ·J Natl Compr Canc Netw · Pubmed #27956542.

ABSTRACT: Myelofibrosis (MF), polycythemia vera (PV), and essential thrombocythemia (ET) are a group of heterogeneous disorders of the hematopoietic system collectively known as Philadelphia chromosome-negative myeloproliferative neoplasms (MPNs). The diagnosis and the management of patients with MPNs have evolved since the identification of mutations that activate the JAK pathway (JAK2, CALR, and MPL mutations) and the development of targeted therapies has resulted in significant improvements in disease-related symptoms and quality of life. This manuscript discusses the recommendations outlined in the NCCN Guidelines for the diagnostic workup of MPN (MF, PV, and ET), risk stratification, treatment, and supportive care strategies for the management of MF.

18 Guideline Light chains removal by extracorporeal techniques in acute kidney injury due to multiple myeloma: a position statement of the Onconephrology Work Group of the Italian Society of Nephrology. 2016

Fabbrini, P / Finkel, K / Gallieni, M / Capasso, G / Cavo, M / Santoro, A / Pasquali, S. ·Clinica Nefrologica, ASST Monza San Gerardo Hospital, University Milano-Bicocca, Osp San Gerardo, via Pergolesi 33, 20851, Milan, Monza, Italy. p.fabbrini@asst-monza.it. · UT Health Science Center, University of Texas, 6410 Fannin St., Ste. 606, Houston, TX, 77030, USA. · Department of Clinical and Biomedical Sciences, Nephrology and Dialysis Unit, San Carlo Borromeo Hospital, University of Milan, Milan, Italy. · Chair of Nephrology, Department of Cardio-Vascular Medicine, Second University of Naples, Naples, Italy. · Department of Experimental, Diagnostic and Specialty Medicine, Hematology/Oncology, Policlinico Sant'Orsola, Bologna, Italy. · Nephrology, Dialysis, and Hypertension Unit, Sant'Orsola-Malpighi University Hospital, Bologna, Italy. · U.O.C Nephrology and Dialysis, S. Maria Nuova Hospital, Reggio Emilia, Italy. ·J Nephrol · Pubmed #27757797.

ABSTRACT: Acute kidney injury (AKI) is a frequent complication of multiple myeloma and is associated with increased short-term mortality. Additionally, even a single episode of AKI can eventually lead to end-stage renal disease (ESRD), significantly reducing quality of life and long-term survival. In the setting of multiple myeloma, severe AKI (requiring dialysis) is typically secondary to cast nephropathy (CN). Renal injury in CN is due to intratubular obstruction from precipitation of monoclonal serum free light chains (sFLC) as well as direct tubular toxicity of sFLC via stimulation of nuclear factor (NF)κB inflammatory pathways. Current mainstays of CN treatment are early removal of precipitating factors such as nephrotoxic drugs, acidosis and dehydration, together with rapid reduction of sFLC levels. Introduction of the proteasome inhibitor bortezomib has significantly improved the response rates in multiple myeloma due to its ability to rapidly reduce sFLC levels and has been referred to as "renoprotective" therapy. As an adjunct to chemotherapy, several new extracorporeal techniques have raised interest as a further means to reduce sFLC concentrations in the treatment of CN. Whether addition of extracorporeal therapies to renoprotective therapy can result in better renal recovery is still a matter of debate and there are currently no guidelines in this field. In this positon paper, we offer an overview of the available data and the authors' perspectives on extracorporeal treatments in CN.

19 Guideline Cytogenetics in the management of multiple myeloma: an update by the Groupe francophone de cytogénétique hématologique (GFCH). 2016

Daudignon, Agnès / Quilichini, Benoît / Ameye, Geneviève / Poirel, Hélène / Bastard, Christian / Terré, Christine. ·UF de cytogénétique hématologique, Pôle biologie-hygiène, Valenciennes, France. · Service de cytogénétique, Laboratoire Biomnis, Lyon, France. · Centre de génétique humaine, Cliniques universitaires St-Luc-Université catholique de Louvain, Bruxelles, Belgique. · Centre de génétique humaine, Cliniques universitaires St-Luc & de Duve Institute-Université catholique de Louvain, Bruxelles, Belgique. · Département de génétique, Centre Henri Becquerel, Rouen, France. · Laboratoire de cytogénétique, Centre de transfusion sanguine, Le Chesnay, France. ·Ann Biol Clin (Paris) · Pubmed #27707673.

ABSTRACT: Cytogenetics of multiple myeloma has evolved in recent years by the emergence of Interphasic fluorescence in situ hybridization (FISH) performed on sorted plasma cells detecting abnormalities independently of a proliferative and infiltrative index. Cytogenetic analysis plays a major part in the risk stratification of myeloma diagnosis due to prognostic impact of various cytogenetic abnormalities as well as to the association between emerging therapeutic approaches in MM. Thus, practice guidelines now recommend interphasic FISH or alternative molecular technics as the initial analysis for multiple myeloma. The Groupe francophone de cytogénétique hématologique (GFCH) proposes in this issue an update of managing multiple myeloma cytogenetics.

20 Guideline Consensus on Surgical Management of Myeloma Bone Disease. 2016

Anonymous2040881. · ·Orthop Surg · Pubmed #27627707.

ABSTRACT: Myeloma bone disease (MBD), the skeletal lesions caused by multiple myeloma, is also known as skeletal related events and includes bone pain, osteoporosis, pathological fractures, osteolytic bone lesions, spinal instability, spinal cord and nerve root compression and extramedullary plasmacytoma. It is now generally accepted that patients with these complications usually require surgical management and that such treatment is safe and effective. The aims of surgical interventions are to alleviate pain, improve quality of life, treat potential or existing pathological fractures, decompress the spinal cord and nerve roots, and reestablish bone continuity. Thus far, there have not been uniform standards for surgical treatment of MBD. The Surgeon's Committee of the Chinese Myeloma Working Group has therefore achieved a consensus with the aim of providing guidance for clinicians and benefitting patients with MBD. This consensus focuses on the treatment of MBD, including its clinical definition and characteristics, diagnosis and surgical management. This expert consensus document was compiled after discussion and revision by experts from several relevant institutions in China. However, it is only an interim guide that cannot be enforced legally. It will be updated with development of new techniques of treatment.

21 Guideline IFM (Intergroupe francophone du myélome) recommendations for uniform interpretation of serum and urine protein electrophoresis in multiple myeloma diagnosis and follow-up. 2016

Dejoie, Thomas / Lakomy, Daniela / Caillon, Hélène / Pegourié, Brigitte / Decaux, Olivier. ·Laboratoire de biochimie spécialisée, CHU de Nantes, France. · Laboratoire de biochimie spécialisée, CHU de Dijon, France. · Service d'hématologie, CHU de Grenoble, France. · Service de médecine interne, CHU de Rennes, France. ·Ann Biol Clin (Paris) · Pubmed #27492696.

ABSTRACT: Serum and urine proteins electrophoresis take a major place in multiple myeloma management, at time of diagnosis, during follow-up for treatment response evaluation and also in detection of relapse. The Intergroupe francophone du myélome (IFM) suggests recommendations to clinicians and biologists, to perform and interpret these biochemical analysis, with the objective of harmonizing practices between laboratories and improving patients' follow-up.

22 Guideline [Mobilization of peripheral blood stem cells with plerixafor in poor mobilizer patients]. 2016

Sancho, Juan-Manuel / Duarte, Rafael / Medina, Laura / Querol, Sergi / Marín, Pedro / Sureda, Anna / Anonymous13210873. ·Servicio de Hematología Clínica, Institut Català d'Oncologia (ICO)-Hospital Germans Trias i Pujol, Institut Josep Carreras para la Lucha contra la Leucemia, Badalona, Barcelona, España. Electronic address: jsancho@iconcologia.net. · Servicio de Hematología, Institut Català d'Oncologia (ICO)-Hospital Duran i Reynals, Hospitalet de Llobregat, Barcelona, España. · Banc de Sang i Teixits de Catalunya, Barcelona, España. · Servicio de Hemoterapia y Hemostasia, Hospital Clínic i Provincial, Barcelona, España. ·Med Clin (Barc) · Pubmed #27374031.

ABSTRACT: BACKGROUND AND OBJECTIVE: Poor mobilization of peripheral blood stem cells (CD34(+) cells) from bone marrow is a frequent reason for not reaching the autologous stem cell trasplantation (SCT) procedure in patients diagnosed with lymphoma or myeloma. Plerixafor, a reversible inhibitor of the binding of stromal cell-derived factor 1 to its cognate receptor CXCR4, has demonstrated a higher capacity for the mobilization of peripheral blood stem cells in combination with granulocyte colony stimulating factor (G-CSF) compared with G-CSF alone. For this reason, plerixafor is now indicated for poor mobilizer myeloma or lymphoma patients. Some studies have recently indicated that a pre-emptive strategy of plerixafor use during first mobilization, according to the number of CD34(+) mobilized cells in peripheral blood or to the harvested CD34(+) cells after first apheresis, could avoid mobilization failures and re-mobilizations, as well as the delay of autologous SCT. The aim of this consensus was to perform a review of published studies on pre-emptive strategy and to establish common recommendations for hospitals in Catalonia and Balearics on the use of pre-emptive plerixafor. METHODS: For the Consensus, physicians from participant hospitals met to review previous studies as well as previous own data about plerixafor use. The GRADE system was used to qualify the available evidence and to establish recommendations on the use of pre-emptive plerixafor. RESULTS AND CONCLUSIONS: After a review of the literature, the expert consensus recommended the administration of pre-emptive plerixafor for multiple myeloma or lymphoma patients with a CD34+ cell count lower than 10 cells/μL in peripheral blood (measured in the morning of day 4 of mobilization with G-CSF or after haematopietic recovery in the case of mobilization with chemotherapy plus G-CSF).

23 Guideline The use of enhanced half-life coagulation factor concentrates in routine clinical practice: guidance from UKHCDO. 2016

Collins, P / Chalmers, E / Chowdary, P / Keeling, D / Mathias, M / O'Donnell, J / Pasi, K J / Rangarajan, S / Thomas, A. ·Arthur Bloom Haemophilia Centre, University Hospital of Wales, Cardiff, UK. · Haemophilia Centre, Royal Hospital for Sick Children, Glasgow, UK. · Katharine Dormandy Haemophilia Centre, Royal Free Hospital, London, UK. · Haemophilia Centre, Churchill Hospital, Oxford, UK. · Haemophilia Centre, Great Ormond Street Hospital, London, UK. · Haemophilia Centre, St. James Hospital, Dublin, Ireland. · Haemophilia Centre, Royal London Hospital, London, UK. · Haemophilia Centre, Basingstoke and North Hampshire Hospital, Basingstoke, UK. · Haemophilia Centre, Royal Infirmary of Edinburgh, Edinburgh, UK. ·Haemophilia · Pubmed #27311929.

ABSTRACT: Enhanced half-life factor VIII and IX products are being introduced into routine clinical practice. Published data report on clinical trials and there are limited data available on how to use these products in routine clinical practice. Many patients, for example, those with a past history of an inhibitor, have been excluded from clinical trials and there are limited data published on children. This guidance document is a consensus statement from the UK Haemophilia Centres Doctors' Organisation and aims to give pragmatic advice on the use of these products in routine practice.

24 Guideline When and how to start prophylaxis in boys with severe hemophilia without inhibitors: communication from the SSC of the ISTH. 2016

Fischer, K / Collins, P W / Ozelo, M C / Srivastava, A / Young, G / Blanchette, V S. ·Van Creveldkliniek, University Medical Center Utrecht, Utrecht, the Netherlands. · Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht, the Netherlands. · Arthur Bloom Haemophilia Centre, School of Medicine, Cardiff University, Cardiff, UK. · INCT do Sangue Hemocentro Unicamp, University of Campinas, Campinas, Brazil. · Faculty of Medical Sciences Unicamp, University of Campinas, Campinas, Brazil. · Department of Haematology, Christian Medical College, Vellore, India. · Hemostasis and Thrombosis Center, Children's Hospital, Los Angeles, CA, USA. · University of Southern California Keck School of Medicine, Los Angeles, CA, USA. · Pediatric Thrombosis and Hemostasis Program, Hospital for Sick Children, University of Toronto, Toronto, Canada. · Department of Pediatrics, University of Toronto, Toronto, Canada. ·J Thromb Haemost · Pubmed #27186714.

ABSTRACT: -- No abstract --

25 Guideline Promoting self-management and adherence during prophylaxis: evidence-based recommendations for haemophilia professionals. 2016

Schrijvers, L H / Schuurmans, M J / Fischer, K. ·Van Creveldkliniek, University Medical Center Utrecht, Utrecht, the Netherlands. · Nursing Science, Faculty of Health Care, University of Applied Science, Utrecht, the Netherlands. · Nursing Science, University Medical Center Utrecht, Utrecht, the Netherlands. · Julius Centre for Health Sciences and Primary Care, University Medical Center, Utrecht, The Netherlands. ·Haemophilia · Pubmed #27075653.

ABSTRACT: INTRODUCTION: Throughout life, a patient with severe haemophilia is confronted with many treatment-related challenges. Insight into self-management and non-adherence could improve the quality of care for these patients. The aim of this study was to provide an overview of the current evidence on self-management and adherence to prophylaxis in haemophilia. METHOD: Based on series of studies and published literature, aspects of treatment were explored: learning and performing self-infusion, achieving self-management skills in adolescence, adherence issues and coping with haemophilia. Evidence-based and age-group-specific recommendations for haemophilia professionals were formulated. RESULTS: Nearly, all severe haemophilia patients and parents were able to perform self-infusion and the quality level of infusion skills was acceptable. Learning self-infusion was generally initiated before the onset of puberty and full self-management was obtained 10 years later. Adherence was defined using a Delphi consensus procedure and was determined by skipping, dosing and timing of infusions. Adherence levels varied according to age, with highest levels in children (1-12 years) and the lowest among 25-40 years. Adherence to prophylaxis was acceptable (43%), yet 57% of the population struggled with prophylaxis. Qualitative research showed that the position of prophylaxis in life is the main driver of adherence. This position is influenced by acceptance and self-management skills. Regarding coping with haemophilia, the majority of patients used a problem-focused approach. CONCLUSION: Self-management and adherence to prophylaxis vary during the life span. Acceptance of the disease and self-management skills were important aspects that may require tailored professional support.

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