Through their clinical medical knowledge base, eMedicine, Medscape offers current, comprehensive information about PNH, from its pathophysiology, etiology, and epidemiology; to typical presentations and differential diagnoses; to patient workup, treatment, and follow-up. There are also Practice Essentials, Questions & Answers, Media Gallery, and References sections.
In May 2021, the National Organization for Rare Disorders (NORD) and the Aplastic Anemia & MDS International Foundation (AAMDSIF) presented this 24-minute webinar. Participants from NORD and AAMDSIF provide background information about resources that their organizations offer to patients and healthcare professionals; review NORD’s IAMRARE® Registry Program and data gathering for patient registries for natural history studies; and explain Global PNH Registry data collection aims and processes for storage and use.
MedlinePlus Genetics, a part of the National Library of Medicine’s consumer resource MedlinePlus, offers patients solid information about the etiology of pyruvate kinase deficiency (and many other genetic disorders), plus vetted resources for genetic testing and appropriate clinical trials.
Patient Worthy, an online advocacy and information website for people with rare diseases, recently launched their Rare Classroom series. Featured in July is a reader-friendly post on PNH, with bulleted descriptions providing essential information about its etiology, causes, symptoms, and treatment, as well as links to relevant newsbriefs, research, and national organizations.
This comprehensive 2018 booklet for patients also provides useful information for healthcare providers who are not specialists in the management of PKD. The publication was independently developed by S. Karger Publishers Limited, with the support of an educational grant from Agios.
The CDMRP, created in 1992, is a partnership between the public, the US Congress, and the military. This February 2021 pamphlet highlights current high-impact BMF clinical research, including a link to summary abstracts and publication citations for studies funded through the Bone Marrow Failure Research Program.
While the management of COVID-19 continues to evolve, these recommendations from the European Society for Blood and Marrow Transplantation provide useful guidance on stem cell transplant (HCT) in the context of COVID-19.
Marrowforums was founded by volunteers for, and board members of, the nonprofit Aplastic Anemia & Myelodysplastic Syndromes International Foundation, as a forum for patients with BMF diseases such as AA, MDS, and PNH and their extended support system (family members, friends and caregivers, healthcare professionals). In addition to hosting discussion groups, the website provides links to medical information, clinical trials, and patient advocacy resources.
The goal of 30-year-old Be the Match is to connect patients with donor matches for life-saving adult bone marrow or umbilical cord blood transplants. Be the Match is operated by the National Marrow Donor Program® (NMDP), a nonprofit organization that matches patients with donors, educates healthcare professionals, and conducts research through its Center for International Blood and Marrow Transplant Research®. In an April 2021 news release, the organization noted that since its inception, it has facilitated more than 105,000 transplants for patients with leukemia, lymphoma, sickle cell disease, and approximately 70 other diseases.
The NIDDK website offers user-friendly sections including Definition & Facts; Symptoms & Causes; Diagnosis; Treatment; Eating, Diet, & Nutrition; and Clinical Trials. Information about related conditions and diseases, as well as related research into hematologic diseases at NIDDK, is also provided.
The European School of Haematology, or ESH, held its first translational research e-conference on bone marrow failure disorders live online from November 13–15, 2020. Peruse the website for free access to e-posters, as well as detailed study summaries in the 142-page online scientific program book.
Because inherited bone marrow failure (IBMF) syndromes are associated with a high risk of cancer, researchers and clinicians at the National Cancer Institute have studied patients with IBMF for several decades, and have recently uncovered several new genetic causes of IBMF. The NCI Inherited Bone Marrow Failure Syndrome Cohort Study enrolls families with one or more members with an IBMF syndrome, and includes first-degree and other relatives as appropriate.
The authors of this StatPearls e-book focus on inherited bone marrow failure (BMF) disorders, with sections including but not limited to etiology and pathophysiology, evaluation and management, differential diagnoses, patient education, and clinical pearls related to several common BMF syndromes.
The website of this highly rated charitable organization provides education (patient guides, fact sheets, research summaries) and advocacy resources related to benign hematologic malignancies including aplastic anemia, paroxysmal nocturnal hemoglobinuria, and related bone marrow failure disorders.