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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.

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: -Combination...

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...

Gene Mutations in Aplastic Anemia

Originally Published: 01/12/2015
Note: This review is based upon a presentation at the 2014 American Society of Hematology (ASH) Annual Meeting, December 6 - 9 in San Francisco, CA.The full abstract may be reviewed on the ASH Annual Meeting Web site.  Tetsuichi Yoshizato, M.D.; Bogdan Dumitriu, M.D.; Kohei Hosokawa, M.D., Ph.D.; Hideki Makishima, M.D., Ph.D.; Kenichi Yoshida, M.D., Ph.D.; Aiko Sato, Ph.D.; Yusuke Okuno, M.D., Ph.D.; Keisuke Kataoka, M.D., Ph.D.; Kenichi Chiba, Ph.D.; Hiroko Tanaka, Yuichi Shiraishi, Ph.D.; Yasunobu Nagata, M.D., Ph.D.; Hiromichi Suzuki, M.D.; Yusuke Sato, M.D.; Yusuke Shiozawa, M.D.;...

Immune System Suppression and Bone Marrow Failure

Originally Published: 01/12/2015
Note: This review is based upon a presentation at the 2014 American Society of Hematology (ASH) Annual Meeting, December 6 - 9 in San Francisco, CA.The full abstract may be reviewed on the ASH Annual Meeting Web site.  Neal S. Young, M.D.; Bogdan Dumitriu, M.D.; and Seishi Ogawa, M.D., Ph.D. SCI-21: Acquired Aplastic Anemia:  New Genetics, New Genomics T lymphocytes are white blood cells that help protect the body from infection. In aplastic anemia, T lymphocytes destroy both immature blood cells and cells that form blood cells. Treatments that suppress the immune system (...

Role of microRNAs in Aplastic Anemia

Originally Published: 01/12/2015
Note: This review is based upon a presentation at the 2014 American Society of Hematology (ASH) Annual Meeting, December 6 - 9 in San Francisco, CA.The full abstract may be reviewed on the ASH Annual Meeting Web site.  Kohei Hosokawa, M.D., Ph.D.; Pawel Muranski, M.D.; Xingmin Feng, Ph.D.; Keyvan Keyvanfar; Danielle M. Townsley, M.D.; Bogdan Dumitriu, M.D.; Jichun Chen, Ph.D.; Sachiko Kajigaya, Ph.D.; James G. Taylor, M.D.; Christopher S. Hourigan, M.D., Ph.D.; John Barrett, M.D.; and Neal S. Young, M.D.   Abstract 2940: Altered microRNAs in T cells from Patients with Acquired Aplastic...