{
  "abstract": "Background: Autologous stem cell transplantation (ASCT) remains the standard consolidative therapy for transplant-eligible patients with multiple myeloma. However, the clinical benefit observed in randomized trials may not be fully reproduced in routine practice because transplant-related mortality (TRM) varies across transplant centers. We developed a mathematical model to quantify how center-specific TRM influences the effective benefit of ASCT. Aim: To develop and validate a mathematical model estimating the impact of excess transplant-related mortality on the expected clinical benefit of upfront ASCT in multiple myeloma. Methods: The Transportability-Based Risk-Adjusted Benefit Model (TRABM) was developed using a competing-risk approach in which early transplant-related death prevents patients from achieving the survival benefit of ASCT. The model incorporates three variables: absolute treatment benefit (Δ), baseline outcome in the control group (B₀), and excess transplant-related mortality (p), generating estimates of adjusted net benefit and treatment transportability. Model parameters were derived from pooled phase III trials (IFM 2009, DETERMINATION, EMN02/HO95, and FORTE), corresponding to an estimated progression-free survival gain of 16.5 months over a baseline of 32 months. Sensitivity analyses evaluated excess TRM from 0.5% to 10%. Results: Increasing excess TRM produced a progressive, nearly linear reduction in the net clinical benefit of ASCT. The estimated progression-free survival gain declined from 16.3 months at 0.5% excess mortality to 16.0 months at 1%, 15.5 months at 2%, 14.1 months at 5%, and 11.6 months at 10%. More than 95% of the benefit observed in randomized trials was preserved when excess TRM remained close to 1%, whereas clinically meaningful attenuation occurred with higher mortality rates. Simulations also demonstrated that patients with greater baseline treatment benefit maintained favorable outcomes despite modest increases in TRM, whereas lower-benefit scenarios lost clinical advantage earlier. Conclusion: The TRABM demonstrates that the effectiveness of ASCT depends not only on treatment efficacy but also on the quality and safety of transplant programs. Even modest increases in transplant-related mortality substantially reduce the benefit expected from clinical trials. This mathematical framework provides an objective tool for translating trial results into real-world practice and may support benchmarking of transplant centers, quality improvement initiatives, and individualized decision-making in hematopoietic stem cell transplantation.",
  "abstract_shown": true,
  "affiliations": [
    "Centro de Hematologia de Ribeirão Preto, Ribeirão Preto, SP, Brazil",
    "Faculdade de Ciencias Médicas de São José dos Campos, (Humanitas), São José dos Campos, SP, Brazil; Centro de Hematologia do Vale, São José dos Campos, SP, Brazil"
  ],
  "article_type": null,
  "arxiv": null,
  "author_total": 2,
  "authors": [
    {
      "affs": [
        0
      ],
      "name": "Sebastião José ISMAEL"
    },
    {
      "affs": [
        1
      ],
      "name": "Fernando CALLERA"
    }
  ],
  "bibtex": "https://research.draconitic.com/paper/0twa8x104ttt4.bib",
  "cited_by": 0,
  "doi": "10.1016/j.htct.2026.107923",
  "fulltext_url": "http://www.sciencedirect.com/science/article/pii/S2531137926016706",
  "id": "0twa8x104ttt4",
  "issns": [
    "2531-1379",
    "2531-1387"
  ],
  "landing_url": "https://doi.org/10.1016/j.htct.2026.107923",
  "language": "en",
  "licence": "CC-BY",
  "licence_label": "CC BY",
  "licence_source": "journal",
  "licence_url": "https://creativecommons.org/licenses/by/4.0/",
  "metadata_licence": null,
  "native_subjects": [
    [
      "lcc",
      "RC633-647.5"
    ],
    [
      "openalex",
      "2720|Multiple Myeloma Research and Treatments"
    ]
  ],
  "openalex": "W7223550657",
  "pdf_url": null,
  "pmcid": null,
  "pmid": null,
  "published": "2026-10-23",
  "publisher": "Elsevier",
  "ris": "https://research.draconitic.com/paper/0twa8x104ttt4.ris",
  "sources": [
    {
      "first_seen": "2026-10-30T13:43:35Z",
      "native_id": "9d0948d3b0ba45f1a0b95447c1a20364",
      "source": "doaj",
      "url": "https://doi.org/10.1016/j.htct.2026.107923"
    }
  ],
  "subjects": [
    {
      "field": "medicine",
      "origin": "lcc:RC633-647.5",
      "subfield": "endocrinology-metabolism"
    }
  ],
  "title": "QUANTIFYING THE IMPACT OF TRANSPLANT-RELATED MORTALITY ON THE CLINICAL BENEFIT OF AUTOLOGOUS STEM CELL TRANSPLANTATION: A MATHEMATICAL MODEL OF TREATMENT TRANSPORTABILITY",
  "title_text": "QUANTIFYING THE IMPACT OF TRANSPLANT-RELATED MORTALITY ON THE CLINICAL BENEFIT OF AUTOLOGOUS STEM CELL TRANSPLANTATION: A MATHEMATICAL MODEL OF TREATMENT TRANSPORTABILITY",
  "type": "peer-reviewed",
  "updated_at": "2026-10-30T13:43:35Z",
  "url": "https://research.draconitic.com/paper/0twa8x104ttt4",
  "venue": "Hematology, Transfusion and Cell Therapy",
  "versions": []
}