Newsstand | Page 19 | Aplastic Anemia and MDS International Foundation

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.

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...

Identification of genetic abnormalities in MDS patients that may lead to a better diagnosis and treatment strategy of MDS patients

Originally Published: 03/29/2013
Myelodysplastic syndrome (MDS) represents a group of diseases that can be subdivided into various categories based on clinical, pathological and certain genetic abnormalities.  Subclassification of MDS, especially in terms of predicted risk for a good or bad outcome, is important because it helps doctors to design a treatment plan. New technologies that can search a patient’s complete genetic information for abnormalities in its code (i.e., DNA) have recently revealed additional genetic abnormalities than can be found on a regular basis in blood cells of MDS patients.  This article reviews...

Myelodysplastic Syndromes are a collection of diseases with common, but also different characteristics

Originally Published: 03/29/2013
Myelodysplastic syndrome (MDS) is not a disease that follows a standard path; especially time of survival after diagnosis and the risk to develop leukemia is highly variable among patients. It is therefore believed that MDS represents not one but a group of diseases.  Indeed, there are several systems in place that allow doctors to classify MDS into particular subtypes based on a pre-determined set of diagnostic criteria; there is the French-American-British (FAB) and the World Health Organization (WHO) classification.  These classification systems describe the cellular nature and genetic...

Even "Moderate" Dose Cyclophosphamide for Severe Aplastic Anemia Is Associated with Significant Toxicities and Does Not Prevent Relapse and Clonal Evolution

Originally Published: 02/25/2013
Note: This review is based upon two presentations at the 2012 American Society of Hematology (ASH) Annual Meeting, December 7-10 in Atlanta, Georgia. The full abstracts may be viewed 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. Mutations in genes that are key to the telomerase complex can lead to a variety of disorders, including bone marrow failure and liver and lung fibrosis. The lab led by Neal Young at the NHLBI presented two studies at the 2012 American Society of Hematology Meeting in...

Improved Outcome of Reduced Toxicity Fludarabine, Cyclophosphamide Plus Thymoglobulin Conditioning Regimen for Unrelated Donor Transplantation in Severe Aplastic Anemia: Comparison of Two Multicenter Prospective Studies

Originally Published: 02/25/2013
Note: This review is based upon two presentations at the 2012 American Society of Hematology (ASH) Annual Meeting, December 7-10 in Atlanta, Georgia. The full abstracts may be viewed 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. Mutations in genes that are key to the telomerase complex can lead to a variety of disorders, including bone marrow failure and liver and lung fibrosis. The lab led by Neal Young at the NHLBI presented two studies at the 2012 American Society of Hematology Meeting in...