Newsstand | Page 17 | 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.

Personalized medicine for MDS

Originally Published: 11/19/2015
Research Review: Myelodysplastic syndromes (MDS) are a complex, heterogeneous disorder of the bone marrow.  The disease behaves in a wide spectrum, manifesting in an indolent manner in some patients as opposed to aggressively in others.  Therefore, treatment decisions have to be tailored to the individual patient.  Age and concurrent medical problems also play a role in determining therapy.  Key-Findings: -The International Prognostic Scoring System (IPSS) helps to risk stratify MDS patients into “lower-risk” disease, where improvement in blood counts is the main goal of therapy, and “...

EHA S826: Eltrombopag Added to Standard Immunosuppression as First Treatment in Severe Aplastic Anemia

Originally Published: 10/16/2015
Danielle Townsley, Bogdan Dumitriu, Phillip Scheinberg, Ronan Desmond, Xingmin Feng, Olga Rios, Barbara Weinstein,Janet Valdez, Thomas Winkler, Keyvan Keyvanfar, Stephanie Sellers, Marie Desierto, Sanaz Soltani, Harshraj Leuva, Swetha Valluri,Colin Wu, Katherine Calvo, Andre Larochelle, Cynthia Dunbar, Neal Young The standard first treatment for aplastic anemia is immunosuppressive treatment. This treatment improves blood cell counts in about 60% to 65% of patients with severe aplastic anemia. But blood counts return to normal (known as a complete response) in only about 10% of these...

Incidence and Burden of the Myelodysplastic Syndromes

Originally Published: 10/14/2015
The incidence of myelodysplastic syndromes (MDS) has been underestimated in population based registries due to lack of reporting and under-diagnosis.  Using Medicare databases, the incidence in patients 65 years or older is estimated to be between 75 and 162 per 100,000 and growing.  There are several unmet needs in this patient population, including patients not receiving therapies to potentially reduce transfusion needs and lack of treatment options for patients who fail standard therapy.  In addition, older MDS patients are not always given the opportunity to participate in shared...

AA&MDSIF announces PNH grant awards

Originally Published: 09/20/2015
Earlier this summer, AA&MDSIF called for research proposals addressing Paroxysmal Nocturnal Hemoglobinuria (PNH), made available through a generous donation by the Geczik family. Based on scientific review by an ad-hoc panel of PNH experts, AA&MDSIF selected three proposals to receive funding. Robert A. Brodsky, MD., Johns Hopkins University, will receive an award for his project titled Preclinical Development of an Oral Factor D Complement Inhibitor to Treat PNH. PNH is a life-threatening hemolytic anemia that results from the loss of cell surface complement regulators. These...

The doctor-patient interface: perception of health status and treatment preferences, as well as tools to facilitate communication

Originally Published: 09/15/2015
Communication is a cornerstone of the doctor-patient relationship. Consider the initial encounter that comes with the MDS diagnosis. Out of the time allotted for a consultation, no more than 10 minutes is consumed by the physical exam, paperwork, or other activates related the visit, whereas the vast majority of the time is dedicated to a conversation about the events leading to the patient’s diagnosis, followed by a discussion of what that diagnosis implies, prognosis, and treatment options. Yet, as the Irish playwright George Bernard Shaw put it, “The single biggest problem in...

Clinical Utility of Lenalidomide in the Treatment of Myelodysplastic Syndromes

Originally Published: 06/17/2015
This article reviews lenalidomide in the setting of myelodysplastic syndromes (MDS). Lenalidomide, a thalidomide analog, is an immunomodulatory medication that is used in the lower-risk and intermediate-risk MDS settings and a specific genetic mutation (deletion in the long arm of chromosome 5, abbreviated as del(5q). Thalidomide is no longer used to treat MDS due to its side effects. The mutation of del(5q) is important as it likely deletes genes that are important for erythropoiesis (production of red blood cells) and cell cycle regulation. Genes that are near this del(5q) region include...

Eltrombopag in aplastic anemia

Originally Published: 05/21/2015
Acquired aplastic anemia is an immune-mediated bone marrow failure in most cases. Its treatment is based on immunosuppressive strategies, whether via allogeneic bone marrow transplantation (BMT), providing immunosuppression (through high dose chemotherapy) plus healthy hematopoietic progenitor cells; or with lymphotoxic drugs, such as ATG and cyclosporine. Whereas BMT yields excellent results, with survival rates as high as 70-90%, immunosuppressive therapy (IST) fails to improve hematologic counts in up to one-third of cases, even when potent drugs are added to standard horse ATG plus...

Pathogenesis of MDS: An overview of molecular and non-molecular aspects of the disease

Originally Published: 05/12/2015
Research Review: The myelodysplastic syndromes (MDS) are a bone marrow failure disorder characterized by ineffective maturation of cells, low blood counts, and risk of transformation to acute myeloid leukemia (AML).  The average age of onset is late 60s to early 70s.  There are currently three FDA-approved medications for MDS: lenalidomide, azacitidine, and decitabine.  Although these agents can be effective in the treatment of MDS, none are curative.  The only potential curative therapy is a stem cell transplant.  It is known that genetic/chromosomal instability can contribute to...

More is better: Combination therapies for myelodysplastic syndromes

Originally Published: 04/07/2015
Research Review: The myelodysplastic syndromes are a bone marrow failure disorder characterized by ineffective maturation of cells, low blood counts, and risk of transformation to acute myeloid leukemia (AML).  There are currently three FDA-approved medications for MDS: lenalidomide, azacitidine, and decitabine.  Although these agents can be effective in the treatment of MDS, none are curative.  Much of the current research in the field focuses on combining these active agents with other classes of drugs in the hopes of better efficacy without sacrificing safety. Key-Findings: -...

Telomere Length and Allogeneic Hematopoietic Cell Transplantation for Severe Aplastic Anemia

Originally Published: 04/07/2015
Telomeres are long stretches of DNA associated with protein complexes located at each chromosome end that protect our genetic data.  Each time a cell divides the telomeres shorten and when it is too abbreviated the cell stops proliferating. Consequently, telomere length is a marker of cellular replicative capacity, senescence, and aging. Short telomere length has been associated with more relapse, evolution to MDS or AML (clonal evolutions) and even with a poorer survival in patients with aplastic anemia treated with immunosuppressive therapies, the standard treatment for those who lack a...