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We describe a case of acute myeloid leukemia with NPM1 mutation and disseminated leukemia cutis in a very old patient, who achieved a long-lasting response to the azacitidine/venetoclax combination with molecular complete remission, given the potential value of this rarely observed clinical outcome.
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http://dx.doi.org/10.1159/000531101 | DOI Listing |
Leukemia
September 2025
Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, GA, USA.
Pediatric acute myeloid leukemia (pAML) is a heterogeneous malignancy driven by diverse cytogenetic mutations. While identification of cytogenetic lesions improved risk stratification, prognostication remains inadequate with 30% of standard-risk patients experiencing relapse within 5 years. To deeply characterize pAML heterogeneity and identify poor outcome-associated blast cell profiles, we performed an analysis on 708,285 cells from 164 bone marrow biopsies of 95 patients and 11 healthy controls.
View Article and Find Full Text PDFLeukemia
September 2025
I.R.C.C.S Santa Lucia Foundation, Via del Fosso di Fiorano, Rome, Italy.
At present there is no metabolic characterization of acute promyelocytic leukemia (APL). Pathognomonic of APL, PML::RARα fusion protein rewires metabolic pathways to feed anabolic tumor cell's growth. All-trans retinoic acid (ATRA) and arsenic trioxide (ATO)-based therapies render APL the most curable subtype of AML, yet approximately 1% of cases are resistant and 5% relapse.
View Article and Find Full Text PDFClin Lymphoma Myeloma Leuk
August 2025
The Mikael Rayaan Foundation Global Transplantation and Cellular Therapy Consortium, Kansas City, KS; Division of Hematologic Malignancies & Cellular Therapeutics, University of Kansas Medical Center, Kansas City, KS; U.S Myeloma Innovations Research Collaborative, Kansas City, KS. Electronic addres
Background: Allogeneic hematopoietic stem cell transplantation (allo-HCT) is a key treatment for acute myeloid leukemia (AML). Measurable residual disease (MRD) predicts post-transplant outcomes. This study evaluates the impact of pretransplant MRD status on outcomes in AML patients undergoing allo-HCT.
View Article and Find Full Text PDFTransplant Cell Ther
September 2025
Department of Hematology, the First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China; Zhejiang Provincial Clinical Research Center for Hematological disorders, Hangzhou, China; Zhejiang Key Laboratory for Precision Diagnosis and Treatment of Hematological Maligancies, Han
Background: Haploidentical hematopoietic stem cell transplantation (haplo-HSCT) offers curative potential for hematologic malignancies but is often limited by high incidences of graft-versus-host disease (GVHD), delayed engraftment, and transplant-related mortality-especially when donors are aged ≥40 years. Umbilical cord blood (UCB) infusion may mitigate these risks by promoting immune tolerance and hematopoietic recovery. However, the efficacy of this strategy in the context of older donors remains insufficiently studied.
View Article and Find Full Text PDFTransplant Cell Ther
September 2025
Department of Hematology, Toranomon Hospital, Tokyo, Japan; Okinaka Memorial Institute for Medical Research, Tokyo, Japan.
Background: Umbilical cord blood transplantation (UCBT) is a valuable treatment option with the potential for curative outcomes in patients with myeloid malignancies in non-remission status, but relapse and early non-relapse mortality (NRM) remain significant barriers. Tacrolimus and mycophenolate mofetil (MMF) are widely used as graft-versus-host disease (GVHD) prophylaxis in UCBT, but there is no consensus on the appropriate MMF dose for GVHD prophylaxis.
Objectives: We conducted a retrospective analysis to investigate the impact of MMF dose on outcomes in patients undergoing UCBT at our institution.