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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.

Major Health Care Professional Organizations Call for COVID-19 Vaccine Mandates for All Health Workers

Originally Published: 07/26/2021
Groups include American Medical Association, American Nurses Association, American Academy of Pediatrics, Association of American Medical Colleges, and National Association for Home Care and Hospice (July 26, 2021) - With COVID-19 case counts rising amid the spread of the Delta variant, more than 50 health care professional societies and organizations called for all health care employers to require their employees to be vaccinated against COVID-19 in a joint statement released today. These societies and organizations represent millions of workers throughout health and long-term care – from...

FDA approves belumosudil for chronic graft-versus-host disease

Originally Published: 07/16/2021
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 of systemic therapy. Efficacy was evaluated in KD025-213 (NCT03640481), a randomized, open-label, multicenter dose-ranging trial that included 65 patients with chronic GVHD who were treated with belumosudil 200 mg taken orally once daily. The main efficacy outcome measure was overall response rate (ORR) through...

New target to prevent leukaemia in Down’s syndrome children

Originally Published: 07/16/2021
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 children with Down’s syndrome, and even have potential to transform cancer care in other children. Children with Down’s syndrome have a 150-fold increased risk of developing myeloid leukaemia in their first five years of life. However, the mechanism by which the extra copy of chromosome 21 leads to leukaemia was...

Autoimmune- and complement-mediated hematologic condition recrudescence following SARS-CoV-2 vaccination

Originally Published: 07/15/2021
Key Points Severe exacerbation of underlying hematologic conditions can occur within 1 to 4 days after dose 2 of a 2-dose SARS-CoV-2 vaccine series. A mild exacerbation after dose 1 and/or a history of vaccine-related adverse events may portend a more serious event after dose 2. Abstract A variety of autoimmune disorders have been reported after viral illnesses and specific vaccinations. Cases of de novo immune thrombocytopenia (ITP) have been reported after SARS-CoV-2 vaccination, although its effect on preexisting ITP has not been well characterized. In addition, although COVID-19 has been...

Outcomes in patients with newly diagnosed TP53-mutated acute myeloid leukemia with or without venetoclax-based therapy

Originally Published: 06/28/2021
Abstract Background Venetoclax (VEN) in combination with a hypomethylating agent (HMA) has become the standard of care for patients aged >75 years and for those not eligible for intensive chemotherapy who have newly diagnosed acute myeloid leukemia (AML). The benefit of VEN-based therapy in patients who have newly diagnosed AML with mutations in the TP53 gene (TP53mut) over standard therapy is undefined. Methods In this single-institutional, retrospective analysis, the authors assessed the clinical outcomes of 238 patients with newly diagnosed TP53mut AML and compared the clinical...

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

Originally Published: 06/24/2021
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 Plasmacytoid Dendritic Cell Neoplasm (BPDCN) Program in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Pemmaraju, here. (Watch video at link above)

Pediatric Myelodysplastic Syndromes

Originally Published: 06/24/2021
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 repeat marrow sampling and correlation with ancillary laboratory testing. •Allogeneic hematopoietic stem cell transplant represents the only curative intervention, and should be considered in all patients based on clinical and genetic features. •Comprehensive next-generation sequencing–based studies are starting to...

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

Originally Published: 06/22/2021
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-2 “variants of concern” appearing around the world that could find ways to evade acquired immunity, increasing the chances of new outbreaks. Now, a new NIH-supported study shows that the answer to this question will vary based on how an individual’s antibodies against SARS-CoV-2 were generated: over the course of a...

Core binding factor acute myelogenous leukemia-2021 treatment algorithm

Originally Published: 06/16/2021
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 significantly through the effective implementation of available therapeutic measures and appropriate disease monitoring. The incorporation of gemtuzumab ozogamicin into frontline therapy should be standard. Cytarabine based induction/consolidation regimen may be combined with anthracycline (3 + 7 standard) or antimetabolite...

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

Originally Published: 06/07/2021
 – 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--(BUSINESS WIRE)--Jun. 7, 2021-- Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced the U.S. Food and Drug Administration (FDA) has approved the expanded use of ULTOMIRIS® (ravulizumab-cwvz) to include children (one month of age and older) and adolescents with paroxysmal nocturnal hemoglobinuria (PNH)....