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.
Trends in Volumes and Survival After Hematopoietic Cell Transplantation in Racial/Ethnic Minorities
Originally Published: 04/25/2024
Article Source: External Web Content
Key Points
The number of autoHCT and alloHCT grew faster in Non-Hispanic African Americans and Hispanics compared to Non-Hispanic Whites.
Survival after autoHCT and alloHCT improved over time for all racial/ ethnic groups, though African Americans have worse outcomes.
There has been an increase in volume as well as improvement in overall survival (OS) after hematopoietic cell transplantation (HCT) for hematologic disorders. It is unknown if these changes have impacted racial/ethnic minorities equally. In this observational study from Center for International Blood and Marrow Transplant...
Alternative donor transplantation for severe aplastic anemia: a comparative study of the SAAWP EBMT
Originally Published: 04/21/2024
Article Source: External Web Content
Key Points
SCT from MUD offers superior survival outcomes for severe aplastic anemia compared to both MMUD and Haplo.
The decision between a MMUD or Haplo donor in the absence of an available MUD remains uncertain.
Selecting the most suitable alternative donor becomes challenging in severe aplastic anemia (SAA) when a matched sibling donor (MSD) is unavailable. We compared outcomes in SAA patients undergoing SCT from matched unrelated donors (MUD, n=1106), mismatched unrelated donors (MMUD, n=340), and haploidentical donors (Haplo, n=206) registered in the EBMT database (2012-2021). For...
Comparison of Haploidentical Stem Cell Transplantation with Post-Transplantation Cyclophosphamide versus Umbilical Cord Blood Transplantation in Adult Patients with Aplastic Anemia
Originally Published: 09/18/2023
Article Source: External Web Content
Highlights
Haploidentical stem cell transplantation with post-transplantation cyclophosphamide (PTCy-haplo) and umbilical cord blood transplantation (UCBT) achieved similar overall survival in adult patients with aplastic anemia.
The rate of neutrophil engraftment was higher in the PTCy-haplo group than in the UCBT group.
The cumulative incidence of grade II-IV acute graft-versus-host disease was similar in the 2 groups.
Failure-free survival in patients age <40 years was higher in the PTCy-haplo group.
ABSTRACT
Aplastic anemia patients who are refractory to immunosuppressive therapy or...
Health-Related Quality of Life and Vulnerability among People with Myelodysplastic Syndromes: A US National Study
Originally Published: 05/03/2023
Article Source: External Web Content
Key Points
In MDS, health-related quality of life (HRQoL) was worse for vulnerable participants and those with worse prognosis.
Lower-risk MDS was associated with better HRQoL, but this relationship was lost among the vulnerable.
Health-related quality of life (HRQoL) and vulnerability are variably affected in patients with myelodysplastic syndromes (MDS) and other cytopenic states; however, the heterogeneous composition of these diseases has limited our understanding of these domains. The NHLBI-sponsored MDS Natural History Study (NCT02775383) is a prospective cohort enrolling patients...
Real-World Validation of Molecular International Prognostic Scoring System for Myelodysplastic Syndromes
Originally Published: 03/17/2023
Article Source: External Web Content
ABSTRACT
PURPOSE
Myelodysplastic syndromes (MDS) are heterogeneous myeloid neoplasms in which a risk-adapted treatment strategy is needed. Recently, a new clinical-molecular prognostic model, the Molecular International Prognostic Scoring System (IPSS-M) was proposed to improve the prediction of clinical outcome of the currently available tool (Revised International Prognostic Scoring System [IPSS-R]). We aimed to provide an extensive validation of IPSS-M.
METHODS
A total of 2,876 patients with primary MDS from the GenoMed4All consortium were retrospectively analyzed.
RESULTS
IPSS-M improved...
Blood test identifies acute myeloid leukemia patients at greater risk for relapse after bone marrow transplant
Originally Published: 03/07/2023
Article Source: External Web Content
A small portion of adults in remission from a deadly blood cancer had persisting mutations detected, which predicted their risk of death from having the cancer return
Researchers at the National Institutes of Health show the benefits of screening adult patients in remission from acute myeloid leukemiaexternal link (AML) for residual disease before receiving a bone marrow transplant. The findings, published in JAMAexternal link, support ongoing research aimed at developing precision medicine and personalized post-transplant care for these patients.
About 20,000 adults in the United States are...
New CEO appointed to lead the Aplastic Anemia and MDS International Foundation
Originally Published: 11/30/2022
Article Source: Press Release
FOR IMMEDIATE RELEASE
The Aplastic Anemia and MDS International Foundation
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New CEO appointed to lead the Aplastic Anemia and MDS International Foundation
Bethesda, MD, (November 30, 2022) -- The Board of Directors of the Aplastic Anemia and MDS International Foundation (AAMDSIF) is pleased to announce the appointment of Brad A. Wong as Chief Executive Officer and Executive Director. Starting December 5, 2022, Mr. Wong will take the reins of the world's leading nonprofit health organization dedicated to supporting patients and...
Effective treatment of low risk acute GVHD with itacitinib monotherapy
Originally Published: 09/12/2022
Article Source: External Web Content
Key Points
Itacitinib monotherapy is as effective as systemic corticosteroids for the treatment of low risk acute GVHD.
Itacitinib monotherapy resulted in fewer serious infections compared to systemic corticosteroids.
The standard primary treatment for acute graft vs host disease (GVHD) requires prolonged, high dose systemic corticosteroids (SCS) that delay reconstitution of the immune system. We used validated clinical and biomarker staging criteria to identify a group of patients with low risk (LR) GVHD that is very likely to respond to SCS. We hypothesized that itacitinib, a selective...
Summer MDS Update 2022
Originally Published: 08/18/2022
Article Source: MDS Update
Press Release Gamida Cell Announces FDA Acceptance of Biologics License Application for Omidubicel with Priority Review
Originally Published: 08/01/2022
Article Source: External Web Content
BOSTON--(BUSINESS WIRE)--Aug. 1, 2022-- Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapy candidates for patients with hematologic and solid cancers and other serious diseases, announced today that the U.S. Food and Drug Administration (FDA) has accepted for filing the Company’s Biologics License Application (BLA) for omidubicel for the treatment of patients with blood cancers in need of an allogenic hematopoietic stem cell transplant. Omidubicel is a first-in-class, advanced NAM-enabled stem cell therapy candidate with breakthrough and orphan drug...