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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.

Senate floats another red-herring health bill

Originally Published: 09/19/2017
The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, issued the following statement opposing the “Graham-Cassidy” plan to repeal and replace the Affordable Care Act (ACA): “The Senate is currently considering a proposal put forward by Senators Graham, Cassidy, Heller, and Johnson that would repeal and replace the Affordable Care Act (ACA). If passed, this plan (known as ‘Graham-Cassidy’) has the potential to jeopardize access to care for millions of individuals with rare diseases. Once...

Explaining SCT vs. Cellular Therapies

Originally Published: 09/08/2017
Donor stem cell transplants and other cellular therapies are treatment approaches that harness the immune system to fight cancer using cells from the patient or from healthy donors. Stem cell transplants are used to treat blood-related cancers such as leukemia, lymphoma, and multiple myeloma, as well as certain non-cancerous blood disorders. Patients first receive chemotherapy and/or radiation therapy to erase or reduce the number of cells in their bone marrow, home to the hematopoietic (blood system) stem cells that give rise to all other types of blood cells. They then receive an infusion...

Patient Safety in Clinical Trials

Originally Published: 09/08/2017
Clinical trial participants are a vital part of cancer research. (Video from National Cancer Institute at NIH)

Promising trial results for lower-risk MDS drug

Originally Published: 09/06/2017
Myelodysplastic syndromes are characterised by ineffective erythropoiesis. Luspatercept (ACE-536) is a novel fusion protein that blocks transforming growth factor beta (TGF β) superfamily inhibitors of erythropoiesis, giving rise to a promising new investigative therapy. We aimed to assess the safety and efficacy of luspatercept in patients with anaemia due to lower-risk myelodysplastic syndromes.

New study on how red blood cells mature

Originally Published: 09/05/2017
Every cell in the body, whether skin or muscle or brain, starts out as a generic cell that acquires its unique characteristics after undergoing a process of specialization. Nowhere is this process more dramatic than it is in red blood cells. In order to make as much room as possible for the oxygen-carrying protein hemoglobin, pretty much everything else inside these precursor red blood cells—nucleus, mitochondria, ribosomes and more—gets purged. Jam-packing red blood cells with hemoglobin is essential. Doing so ensures that all the body’s tissues and organs are well nourished with oxygen to...

Study suggests heart drugs may boost chemotherapy in AML

Originally Published: 09/05/2017
Scientists may have identified a way to boost the effect of chemotherapy against one of the most common forms of leukemia in adults: acute myeloid leukemia. In a new study, researchers found that acute myeloid leukemia (AML) triggers blood vessel leakage in bone marrow, which prevents chemotherapy drugs from being effectively delivered to leukemia cells. However, by using drugs normally used to treat heart and blood vessel disease, the researchers found that they were able to prevent this blood leakage and increase the efficacy of chemotherapy. First study author Diana Passaro, of the...

Allogeneic transplant: does age still matter?

Originally Published: 08/31/2017
In this issue of Blood, on behalf of the Center for International Blood and Marrow Transplant Research (CIBMTR), Muffly et al report on the incidence and outcome of allogeneic stem cell transplantations (alloSCTs) performed between 2000 and 2013 in 1106 patients aged ≥70 years. The incidence of alloSCT in this age group accounted for 0.1% of all stem cell transplantations (SCTs) reported to the CIBMTR in 2001, and it rose to 3.85% by 2013. Comparison of 2 time periods of performing SCTs, 2000 to 2007 and 2008 to 2013, revealed significant improvement in overall survival (OS) and progression-...

FDA approves new drug for relapsed or refractory AML

Originally Published: 08/30/2017
On August 1, 2017, the U.S. Food and Drug Administration granted regular approval to enasidenib (IDHIFA, Celgene Corp.) for the treatment of adult patients with relapsed or refractory acute myeloid leukemia with an isocitrate dehydrogenase-2 (IDH2) mutation as detected by an FDA-approved test. This is the first FDA approval for relapsed or refractory AML specifically with an IDH2 mutation. The FDA concurrently approved a companion diagnostic, the RealTime IDH2 Assay, used to detect the IDH2 mutation. The enasidenib approval was based on Study AG221-C-001 (NCT01915498), an open-label, single-...

New treatment approved for certain high-risk AML

Originally Published: 08/30/2017
The U.S. Food and Drug Administration today approved Vyxeos for the treatment of adults with two types of acute myeloid leukemia (AML): newly diagnosed therapy-related AML (t-AML) or AML with myelodysplasia-related changes (AML-MRC). Vyxeos is a fixed-combination of chemotherapy drugs daunorubicin and cytarabine. “This is the first approved treatment specifically for patients with certain types of high-risk AML,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug...

Marrow protein may be key to SCT success

Originally Published: 08/29/2017
Bone marrow contains hematopoetic stem cells, the precursors to every blood cell type. These cells spring into action following bone marrow transplants, bone marrow injury and during systemic infection, creating new blood cells, including immune cells, in a process known as hematopoiesis. A new study led by University of Pennsylvania and Technical University of Dresden scientists has identified an important regulator of this process, a protein called Del-1. Targeting it, the researchers noted, could be an effective way to improve stem cell transplants for both donors and recipients. There...
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