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Newsstand

Here's where you'll find a regularly updated, broad range of articles written by the AAMDSIF team, allied health organizations and news organizations. By staying well-informed, patients and families are practicing a form of self-support that will help them be more effective self-advocates when engaging with health care providers.

Spectrum of Genetic Alterations in Acquired Aplastic Anemia

Originally Published: 02/10/2014
Note: This review is based upon a presentation at the 2013 American Society of Hematology (ASH) Annual Meeting, December 7-10 in New Orleans, Louisiana.The full abstract may be reviewed on the ASH Annual Meeting Web site. Search by entering the title in the search box. The abstract number is referenced to access the full report. Abstract # 2464 Tetsuichi Yoshizato, M.D., Bogdan Dumitriu, M.D., Kohei Hosokawa, M.D., Ph.D., Hideki Makishima, M.D., Ph.D., Kenichi Yoshida, M.D., Yusuke Okuno, Kenichi Chiba, Hiroko Tanaka, Yuichi Shiraishi, Ph.D., Yasunobu Nagata, M.D., Takamasa Katagiri, Ph.D...

Complement Blockade with a C1 Esterase Inhibitor in Paroxysmal Nocturnal Hemoglobinuria

Originally Published: 01/31/2014
Note: This review is based upon a presentation at the 2013 American Society of Hematology (ASH) Annual Meeting, December 7-10 in New Orleans, Louisiana.The full abstract may be reviewed on the ASH Annual Meeting Web site. Search by entering the title in the search box. The abstract number is referenced to access the full report. Abstract # 594 Amy E. DeZern, M.D., M.H.S., Marc Uknis, M.D., Ph.D., Galina Mukhina, M.D., Ph.D., Jo Anne Saye, Ph.D., Jeffrey J. Pu, M.D., Ph.D., and Robert Brodsky, M.D. Patients with paroxysmal nocturnal hemoglobinuria (PNH) don’t have two important proteins,...

Insights into the Natural History of Paroxysmal Nocturnal Hemoglobinuria (PNH): Analysis of the Presenting Clinical, Haematological and Flow Cytometric Features of 705 Patients Leads to Improved Classification and Prediction of Clinical Course

Originally Published: 01/31/2014
Note: This review is based upon a presentation at the 2013 American Society of Hematology (ASH) Annual Meeting, December 7-10 in New Orleans, Louisiana.The full abstract may be reviewed on the ASH Annual Meeting Web site. Search by entering the title in the search box. The abstract number is referenced to access the full report. Abstract # 3718 Stephen John Richards, Ph.D., Richard Kelly, M.D., Anita Hill, M.B.Ch.B., Ph.D., Anita Dickinson, M.Sc., Fiona Cullen, M.Sc., Jane Shingles, Ph.D., Matthew Cullen, M.Sc., and Peter Hillmen, M.B.Ch.B., Ph.D. A group of researchers from Great...

Clinical mutations associated with MDS and their importance for disease outcome

Originally Published: 11/22/2013
Myelodysplastic syndrome (MDS) is not one disease, but represents a group of diseases.  Each group has distinct clinical and pathological abnormalities.  Some groups are associated with a specific genetic abnormality, others with a mixture of genetic abnormalities.  It is important to understand the significance of the individual abnormalities, because it may help doctors to design a better patient-specific treatment plan or develop entirely new drugs based on what we know about a specific mutation in a specific gene. This study analyzed samples from 738 patients: 603 with MDS and 135 with...

Aplastic Anemia Clinical Trial at the NIH

Originally Published: 08/30/2013
Clinical research—to advance diagnostics and to develop better treatments—requires patients, but one big challenge in a rare disease such as aplastic anemia is patient recruitment. If patients don’t agree to be “on protocol”, or if only a few enroll, clinical trials fail or take years to reach full accrual. Patients are often surprised to learn that unless they are treated on a research trial, data concerning their outcome is not utilized for research purposes.  Also often not appreciated is that even experts with huge clinics rely on carefully conducted and interpreted trials to draw...

Eculizumab Protects Against TE and Prolongs Survival in Patients with Paroxysmal Nocturnal Hemoglobinuria: An International PNH Registry Study

Originally Published: 08/30/2013
Note: Two studies from the Global PNH Registry presented at the European Hematology Association annual meeting in June focused on the effectiveness of eculizumab (Soliris®). Abstract EHA18ABSSUB-5522 Gerard Socie, H. Schrezenmeier, P. Muus, J. Szer, A. Urbano-Ispizua, J. Maciejewski, R. Brodsky, M. Bessler, Y. Kanakura, W. Rosse, G. Khursigara, C. Bedrosian, P. Hillmen  Thromboembolisms, or clots that block vessels, are one of the main causes of death in patients with paroxysmal nocturnal hemoglobinuria (PNH). Clinical trials have shown that eculizumab (Soliris®) reduces rates of...

Improved Fatigue and Quality-of-Life in Patients with Paroxysmal Nocturnal Hemoglobinuria During Treatment with Eculizumab: Data from the Global PNH Registry

Originally Published: 08/30/2013
This study was presented at the European Hematology Association annual meeting in June 2013. Abstract EHA18ABSSUB-4921 Petra Muus, H. Schrezenmeier, G. Socié, J. Maciejewski, J. Szer, R Brodsky, A. Urbano-Ispizua, M. Bessler, Y. Kanakura, W. Rosse, G. Khursigara, C. Bedrosian, P. Hillmen  Paroxysmal nocturnal hemoglobinuria (PNH) causes hemolysis, or destruction of some or all of a patient’s red blood cells. This hemolysis and the other symptoms of PNH have a major impact on patients’ quality of life. Eculizumab (Soliris®) can prevent hemolysis in people with PNH. The PNH Registry is a...

Pathophysiology and management of thrombocytopenia in bone marrow failure: possible clinical applications of thrombopoietin receptor agonists in aplastic anemia and myelodysplastic syndromes.

Originally Published: 08/30/2013
Two acquired bone marrow failure syndromes, aplastic anemia and myelodysplastic syndromes (MDS), have in common that one, two, or all three blood cell lineages may be dangerously low. Neutropenia results in infections, anemia in fatigue and exacerbation of heart conditions, and thrombocytopenia in bleeding. Bone marrow transplantation is the only curative treatment for myelodysplasia. Immunosuppression may lead to improvement in aplastic anemia, but bone marrow transplantation is the standard of care for younger patients and those with a good performance status. Not all patients have a...

Myelodysplastic syndromes: What do hospitalists need to know?

Originally Published: 07/31/2013
This article from the Journal of Hospital Medicine summarizes the key information about myelodysplastic syndromes that hospital-based physicians should understand. It is important for general medicine physicians to recognize the signs and symptoms of MDS, which can lead to referral to a hematologist as well as earlier diagnosis and treatment if indicated.   Myelodysplastic syndromes (MDS) represent a blood disorder characterized by ineffective production of cells leading to low blood counts and risk of development of acute myeloid leukemia.  MDS is more common in the elderly, and the...

Prognostic value of telomere attrition in patients with aplastic anemia

Originally Published: 07/13/2013
Treatment and outcomes for patients with severe aplastic anemia (SAA) have improved markedly over past 40 years due to advances in hematopoietic stem cell transplantation (HSCT) and immunosuppressive therapy (IST). Nonetheless, these treatments have limitations such as failure to engraft or graft versus host disease for HSCT and lack of response or clonal evolution to myelodysplastic syndrome (MDS) with IST.  Refinements are needed in our treatment algorithms to determine who is best suited to each treatment to minimize these complications. We hope to improve on outcomes further in SAA...