{
  "abstract": "In patients with myelodysplastic syndromes (MDS) treated with hypomethylating agents (HMA) and proceeding to allogeneic hematopoietic stem-cell transplantation (alloHSCT), pretransplant risk assessment remains dominated by morphologic remission–based frameworks despite uncertain prognostic value in the transplant setting. We retrospectively analyzed 280 HMA-treated patients with MDS undergoing alloHSCT at DFCI and Moffitt between 2013-2021 to evaluate the predictive performance of IWG 2023 response criteria and to develop an alternative framework integrating functional marrow reserve (FMR) with baseline molecular risk. Progression-free survival (PFS) was the primary outcome. Median overall survival (OS) was 52 months, with 5-year PFS and OS of 43% and 48%, respectively. IWG 2023 response categories showed substantial overlap in outcomes, and assessment by composite complete remission status did not discriminate PFS (5-year PFS 46% vs 40%; P = .16). In contrast, preconditioning hemoglobin and platelet counts were strongly associated with outcomes, whereas marrow blasts and absolute neutrophil count were not. Recursive partitioning identified clinically relevant platelet and hemoglobin thresholds, enabling construction of FMR strata with markedly distinct 5-year PFS (70%, 40%, and 24% for favorable, intermediate and poor risk strata, respectively; P < .0001). Baseline IPSS-M independently predicted outcomes (5-year PFS 58%, 45%, and 29%; P = .003) and further refined prognostication when integrated with FMR. In multivariable analysis, the integrated IPSS-M/FMR model remained independently associated with PFS (hazard ration 3.00–8.31 across risk tiers; P < .004) and showed superior discrimination compared with IWG 2023 and classical transplant variables (PFS concordance index 0.676). These findings support an integrated framework that outperforms remission-based criteria for post-alloHSCT risk prediction in HMA-treated MDS.",
  "abstract_shown": true,
  "affiliations": [
    "Section of Medical Oncology and Hematology, Department of Internal Medicine, Yale School of Medicine, New Haven, CT",
    "Harvard T.H. Chan School of Public Health, Boston, MA",
    "Department of Medical Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA",
    "Department of Data Science, Dana-Farber Cancer Institute, Boston, MA",
    "Division of Blood and Marrow Transplantation and Cellular Therapy, Stanford University, Stanford, CA",
    "Department of Malignant Hematology, H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL"
  ],
  "article_type": "research article",
  "arxiv": null,
  "author_total": 32,
  "authors": [
    {
      "affs": [
        0,
        1,
        2
      ],
      "name": "Luis E. Aguirre"
    },
    {
      "affs": [
        1,
        3
      ],
      "name": "Haesook T. Kim"
    },
    {
      "affs": [
        4
      ],
      "name": "Hany Elmariah"
    },
    {
      "affs": [
        2
      ],
      "name": "Stacey M. Frumm"
    },
    {
      "affs": [
        2
      ],
      "name": "Amar H. Kelkar"
    },
    {
      "affs": [
        2
      ],
      "name": "Vincent Ho"
    },
    {
      "affs": [
        2
      ],
      "name": "Mahasweta Gooptu"
    },
    {
      "affs": [
        2
      ],
      "name": "John Koreth"
    },
    {
      "affs": [
        2
      ],
      "name": "Roman M. Shapiro"
    },
    {
      "affs": [
        2
      ],
      "name": "Rizwan Romee"
    },
    {
      "affs": [
        2
      ],
      "name": "Sarah Nikiforow"
    },
    {
      "affs": [
        2
      ],
      "name": "Joseph H. Antin"
    },
    {
      "affs": [
        2
      ],
      "name": "Robert J. Soiffer"
    },
    {
      "affs": [
        2
      ],
      "name": "Shai Shimony"
    },
    {
      "affs": [
        2
      ],
      "name": "Marlise R. Luskin"
    },
    {
      "affs": [
        2
      ],
      "name": "Jacqueline S. Garcia"
    },
    {
      "affs": [
        2
      ],
      "name": "Evan C. Chen"
    },
    {
      "affs": [
        2
      ],
      "name": "Martha Wadleigh"
    },
    {
      "affs": [
        2
      ],
      "name": "Eric S. Winer"
    },
    {
      "affs": [
        2
      ],
      "name": "Richard M. Stone"
    },
    {
      "affs": [
        2
      ],
      "name": "Daniel J. DeAngelo"
    },
    {
      "affs": [
        5
      ],
      "name": "Najla Al Ali"
    },
    {
      "affs": [
        5
      ],
      "name": "David Sallman"
    },
    {
      "affs": [
        5
      ],
      "name": "Andrew Kuykendall"
    },
    {
      "affs": [
        5
      ],
      "name": "Kendra Sweet"
    },
    {
      "affs": [
        5
      ],
      "name": "Jeffrey E. Lancet"
    },
    {
      "affs": [
        5
      ],
      "name": "Eric Padron"
    },
    {
      "affs": [
        5
      ],
      "name": "Onyee Chan"
    },
    {
      "affs": [
        5
      ],
      "name": "Zhuoer Xie"
    },
    {
      "affs": [
        2
      ],
      "name": "Corey S. Cutler"
    },
    {
      "affs": [
        0,
        2
      ],
      "name": "Maximilian Stahl"
    },
    {
      "affs": [
        5
      ],
      "name": "Rami S. Komrokji"
    }
  ],
  "bibtex": "https://research.draconitic.com/paper/8hqmzdmzs05a5.bib",
  "cited_by": 0,
  "doi": "10.1016/j.bneo.2026.100275",
  "fulltext_url": "https://pmc.ncbi.nlm.nih.gov/articles/PMC13735172/",
  "id": "8hqmzdmzs05a5",
  "issns": [
    "2950-3280"
  ],
  "landing_url": "https://doi.org/10.1016/j.bneo.2026.100275",
  "language": "en",
  "licence": "CC-BY-NC-ND-4.0",
  "licence_label": "CC BY-NC-ND 4.0",
  "licence_source": "article",
  "licence_url": "https://creativecommons.org/licenses/by-nc-nd/4.0/",
  "metadata_licence": null,
  "native_subjects": [
    [
      "heading",
      "Myeloid Neoplasia"
    ],
    [
      "openalex",
      "2720|Acute Myeloid Leukemia Research"
    ]
  ],
  "openalex": "W7171394945",
  "pdf_url": null,
  "pmcid": "PMC13735172",
  "pmid": "42991088",
  "published": "2026-07-27",
  "publisher": "The American Society of Hematology",
  "ris": "https://research.draconitic.com/paper/8hqmzdmzs05a5.ris",
  "sources": [
    {
      "first_seen": "2026-11-09T12:20:54Z",
      "native_id": "PMC13735172",
      "source": "pmc",
      "url": "https://doi.org/10.1016/j.bneo.2026.100275"
    }
  ],
  "subjects": [
    {
      "field": "medicine",
      "origin": "openalex:2720|Acute Myeloid Leukemia Research",
      "subfield": "general"
    }
  ],
  "title": "Integrating functional marrow reserve and molecular risk improves prediction of post-transplant outcomes in HMA-treated myelodysplastic syndromes∗†",
  "title_text": "Integrating functional marrow reserve and molecular risk improves prediction of post-transplant outcomes in HMA-treated myelodysplastic syndromes∗†",
  "type": "peer-reviewed",
  "updated_at": "2026-11-09T12:20:54Z",
  "url": "https://research.draconitic.com/paper/8hqmzdmzs05a5",
  "venue": "Blood Neoplasia",
  "versions": []
}