News and Treatment Updates | Page 4 | Aplastic Anemia and MDS International Foundation (AAMDSIF) Return to top.

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.

Science Simplified: What is a Natural History Study?

Originally Published: 01/17/2022
Article Source: External Web Content
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science accessible and empower rare disease community members with scientific knowledge. Dr. Brown has over a decade of experience in neurodevelopmental research and is currently the Scientific Director for TESS Research Foundation. Please reach out to her at tanya@tessfoundation.org if you have questions or comments. Thank you to Tanya Brown, PhD, Research Program Manager, for writing this article about natural...

Eunice S. Wang, MD, on FLT3-Mutated AML: Gilteritinib and Azacitidine for Intensive Induction Chemotherapy–Ineligible Patients

Originally Published: 01/03/2022
Article Source: External Web Content
Eunice S. Wang, MD, of Roswell Park Comprehensive Cancer Center, discusses phase III results showing that gilteritinib and azacitidine led to significantly higher composite complete response rates in patients with newly diagnosed FLT3-mutant acute myeloid leukemia who are ineligible for intensive induction chemotherapy. Overall survival was similar to that of azacitidine alone (Abstract 700).

Leslie S. Kean, MD, PhD, on Bone Marrow Transplantation: Using Abatacept to Prevent Graft-vs-Host Disease

Originally Published: 01/03/2022
Article Source: External Web Content
Leslie S. Kean, MD, PhD, of Dana-Farber/Boston Children's Cancer and Blood Disorders Center, discusses findings from her analysis of the International Blood and Marrow Transplant Research Database, which led to the recent FDA approval of abatacept for the prevention of acute graft-vs-host disease (GVHD) in adult and pediatric patients. The data suggest improved overall survival with the immunosuppressant abatacept in combination with a calcineurin inhibitor and methotrexate following 7/8 HLA–matched unrelated allogeneic hematopoietic stem cell transplantation (Abstract 3912).

Coronavirus (COVID-19) Update: FDA Authorizes First Oral Antiviral for Treatment of COVID-19

Originally Published: 12/22/2021
Article Source: External Web Content
Today, the U.S. Food and Drug Administration issued an emergency use authorization (EUA) for Pfizer’s Paxlovid (nirmatrelvir tablets and ritonavir tablets, co-packaged for oral use) for the treatment of mild-to-moderate coronavirus disease (COVID-19) in adults and pediatric patients (12 years of age and older weighing at least 40 kilograms or about 88 pounds) with positive results of direct SARS-CoV-2 testing, and who are at high risk for progression to severe COVID-19, including hospitalization or death. Paxlovid is available by prescription only and should be initiated as soon as possible...

MDS Update for Q4 2021 - online version

Originally Published: 12/22/2021
Article Source: MDS Update
Please click the link above to find the online version of the MDS Update for Q4 2021.

ASH 2021 Recap: COVID-19 in Patients with Acute Leukemias & Myelodysplasia In Conversation with Dr. Pinkal Desai

Originally Published: 12/21/2021
Article Source: External Web Content
Podcast recording at link above.

ASH 2021 Recap: Multi Omics Sheds New Light on Malignant Transformation from Myeloproliferative Neoplasm to AML

Originally Published: 12/21/2021
Article Source: External Web Content
Link above leads to podcast recording. 

Monitoring Amphiregulin May Be Warranted in Caring for Patients with Acute GVHD

Originally Published: 12/19/2021
Article Source: External Web Content
Amphiregulin (AREG) may represent a useful longitudinal monitoring biomarker for patients with life-threatening acute graft-vs-host disease (GVHD), according to a sample of 2 prospective clinical trials that was presented at the 2021 ASH Annual Meeting. Specifically, patients with a high baseline of AREG are at an increased mortality risk and, therefore, should be monitored consistently during serial assessments. Specifically, in a multivariate analysis for survival, day 28 responses (risk ratio [RR], 9.14; P <.0001) and baseline AREG (>212 pg/mL; RR, 2.72; P <.05) were the only...

Women With AML Have Worse Outcomes With Gemtuzumab Ozogamicin

Originally Published: 12/17/2021
Article Source: External Web Content
Combination gemtuzumab ozogamicin (GO) and cytarabine is no more effective than standard chemotherapy for older adults with previously untreated, standard-risk acute myeloid leukemia (AML), a phase 2/3 trial suggests.  In fact, GO plus cytarabine was associated with worse event-free survival (EFS) in women.  These findings were presented at the 2021 American Society of Hematology (ASH) Annual Meeting by Juliette Lambert, MD, PhD, of Versailles Hospital in France.  (Article continues at link.)

Aspaveli® (pegcetacoplan) approved in EU as orphan drug for treatment of PNH

Originally Published: 12/15/2021
Article Source: External Web Content
Published: Dec 15, 2021       STOCKHOLM, Dec. 15, 2021 /PRNewswire/ -- The first targeted C3 therapy approved in the EU Approval based on results from head-to-head PEGASUS phase 3 study where Aspaveli demonstrated superiority to eculizumab in improving haemoglobin levels1 Aspaveli will have market exclusivity for paroxysmal nocturnal haemoglobinuria (PNH) based on orphan drug designation Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) and Apellis Pharmaceuticals Inc   (Nasdaq: APLS) announced today that the European Commission (EC) has approved Aspaveli® (pegcetacoplan), the first and...