News and Treatment Updates

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.

FDA approves belumosudil for chronic graft-versus-host disease

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On July 16, 2021, the Food and Drug Administration approved belumosudil (Rezurock, Kadmon Pharmaceuticals, LLC), a kinase inhibitor, for adult and pediatric patients 12 years and older with chronic graft-versus-host disease (chronic GVHD) after failure of at least two prior lines…

New target to prevent leukaemia in Down’s syndrome children

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Researchers have mapped for the first time how and where leukaemia develops in children with Down’s syndrome, it has been announced. Researchers at the Princess Margaret Cancer Centre in Toronto, Canada, say their research could eventually help doctors prevent pre-leukaemia in…

Myeloproliferative Neoplasms Defined: What Are ET, PV, and MF?

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What are essential thrombocythemia (ET) , polycythemia vera (PV), and myelofibrosis (MF) exactly? Dr. Naveen Pemmaraju explains how each of these blood disorders manifests along with the symptoms observed in these MPN patients. Dr. Naveen Pemmaraju is Director of the Blastic…

Pediatric Myelodysplastic Syndromes

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Key points •Pediatric myelodysplastic syndrome (MDS) is rare and often develops in the setting of an underlying inherited bone marrow failure syndrome. •Refractory cytopenia of childhood and MDS with excess blasts comprise most cases. •Definitive diagnosis frequently requires…

How Immunity Generated from COVID-19 Vaccines Differs from an Infection

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A key issue as we move closer to ending the pandemic is determining more precisely how long people exposed to SARS-CoV-2, the COVID-19 virus, will make neutralizing antibodies against this dangerous coronavirus. Finding the answer is also potentially complicated with new SARS-CoV…

Core binding factor acute myelogenous leukemia-2021 treatment algorithm

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Abstract Core binding factor acute myelogenous leukemia (CBF-AML), characterized by the presence of either t(8;21) (q22;q22) or inv(16) (p13q22)/t(16;16), is considered good-risk AML in the context of cytarabine based intensive chemotherapy. Still, outcome can be improved…

Alexion Announces FDA Approval of ULTOMIRIS® (ravulizumab-cwvz) for Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH)

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– With this approval, ULTOMIRIS is the first and only medicine approved in the U.S. to treat children and adolescents with PNH – – Approval based on interim results from Phase 3 study showing ULTOMIRIS demonstrated complete terminal complement inhibition through 26 weeks – BOSTON…

Azacitidine Improves RBC-TI Rates in Patients with Lower-Risk MDS

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CC-486—the oral formulation of azacitidine—significantly improved RBC transfusion independence (RBC-TI) rates and induced durable bilineage improvements in patients with lower-risk myelodysplastic syndromes (LR-MDS) and high-risk disease features, according to a phase 3 clinical…

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