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News and Treatment Updates

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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.

Alternative donor transplantation for severe aplastic anemia: a comparative study of the SAAWP EBMT

Originally Published: 04/21/2024
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
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
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
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
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...

Effective treatment of low risk acute GVHD with itacitinib monotherapy

Originally Published: 09/12/2022
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...

Press Release Gamida Cell Announces FDA Acceptance of Biologics License Application for Omidubicel with Priority Review

Originally Published: 08/01/2022
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...

Patients With MPNs Must Be Honest About Their Symptoms, Says Expert

Originally Published: 06/20/2022
It is incredibly important that patients with MPNs inform their health care team of any symptoms they are experiencing, according to an expert from The Mays Cancer Center at UT Health San Antonio MD Anderson Cancer Center. Because patients with essential thrombocythemia (ET) or polycythemia vera (PV) — rare types of blood cancers known as myeloproliferative neoplasms (MPNs) — experience a range of symptoms that can significantly impact their quality of life, it’s important that patients communicate what they’re experiencing with their health care team. Symptoms may include fatigue, symptoms...

Gamida Cell Completes Rolling Biologics License Application Submission to the FDA for Omidubicel

Originally Published: 06/02/2022
June 2, 2022 - Omidubicel is a first-in-class, advanced NAM-enabled stem cell therapy candidate being evaluated as the first potential allogeneic advanced cell therapy donor source for patients with blood cancers in need of a transplant – - Omidubicel has Orphan Drug Designation and Breakthrough Therapy Designation - BOSTON--(BUSINESS WIRE)--Jun. 2, 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, today announced completion of the rolling Biologics License...

Servier Announces FDA Approval of TIBSOVO® (ivosidenib tablets) in Combination with Azacitidine for Patients with Newly Diagnosed IDH1-mutated Acute Myeloid Leukemia

Originally Published: 05/25/2022
BOSTON, May 25, 2022  – Servier, a leader in oncology committed to bringing the promise of tomorrow to the patients we serve, today announced that the U.S. Food and Drug Administration (FDA) approved TIBSOVO® (ivosidenib tablets) in combination with azacitidine for the treatment of patients with newly diagnosed IDH1-mutated acute myeloid leukemia (AML) in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy. TIBSOVO is the first therapy targeting cancer metabolism approved in combination with azacitidine for patients with newly diagnosed...