{
  "abstract": "Severe thalassemia (ST) is a life-threatening condition common in many low- and middle-income countries. Although bone marrow transplantation (BMT) offers a curative option, its accessibility remains limited because of cost and complexity. This study describes a simplified approach to BMT for hemoglobinopathies that is based on relatively common and inexpensive medicines. This study included 176 pediatric patients with transfusion-dependent thalassemia who received a matched sibling BMT between February 2022 and November 2025 across several centers in Asia and the Middle East. A simplified and low-cost transplant protocol was used, using drugs that are widely available and are listed in the World Health Organization Model List of Essential Medicines. The regimen included fludarabine, dexamethasone, busulfan, and cyclophosphamide. granulocyte colony-stimulating factor–primed bone marrow was used as the graft source, and graft-versus-host disease (GVHD) prophylaxis included cyclosporine and methotrexate. Rejection, mixed-chimerism rates, transplant-related mortality, and GVHD-free, overall survival, and disease-free survival composite outcomes were analyzed. Engraftment was obtained in 97.3% of patients. Thalassemia-free survival was 93.4%, and overall survival was 96%, at a median follow-up of 11.3 months. Rates of moderate to severe acute GVHD were low (3.9%) for a GVHD-free and thalassemia-free composite actuarial outcome of 93.4%. Cytomegalovirus (CMV) reactivation was observed in 19.2% of patients and 1 (0.6%) patient progressed to CMV disease. The approach was equally effective in experienced and newly established transplant centers. A simple, safe, and cost-conscious transplant approach using widely available medicines can achieve excellent outcomes for children with ST in low-resource settings.",
  "abstract_shown": true,
  "affiliations": [
    "Sankalp India Foundation, Bangalore, India",
    "Bhagwan Mahaweer Jain Hospital-Sankalp Centre for Pediatric Hematology Oncology and BMT, Bangalore, India",
    "Sankalp-Health1 Centre for Paediatric Hematology Oncology and Blood and Marrow Transplant, Ahmedabad, India",
    "Mahatma Gandhi Medical College and Hospital and South East Asian Institute of Technology, Jaipur, India",
    "Al-Mujtaba Hospital, Karbala, Iraq",
    "Dr Akbar Niazi Teaching Hospital, Islamabad, Pakistan",
    "Hue Central Hospital, Hue, Vietnam",
    "National Children’s Medical Center, Tashkent, Uzbekistan",
    "Jagriti InnoHealth Platforms Pvt Ltd, Bangalore, India",
    "Cure2Children Foundation, Florence, Italy"
  ],
  "article_type": "research article",
  "arxiv": null,
  "author_total": 17,
  "authors": [
    {
      "affs": [
        0,
        1
      ],
      "name": "Mohana Reddy"
    },
    {
      "affs": [
        0
      ],
      "name": "Tejashree Sridhar"
    },
    {
      "affs": [
        0,
        1
      ],
      "name": "Vidhi Mayur"
    },
    {
      "affs": [
        0,
        1
      ],
      "name": "Geetha T. Lathe"
    },
    {
      "affs": [
        0,
        2
      ],
      "name": "Deepa Trivedi"
    },
    {
      "affs": [
        0,
        2
      ],
      "name": "Vaibhav Shah"
    },
    {
      "affs": [
        0
      ],
      "name": "Rakesh Dhanya"
    },
    {
      "affs": [
        0
      ],
      "name": "Lalith Parmar"
    },
    {
      "affs": [
        3
      ],
      "name": "Priya Marwah"
    },
    {
      "affs": [
        4
      ],
      "name": "Hayder Shahad Marzook"
    },
    {
      "affs": [
        5
      ],
      "name": "Aliya Batool"
    },
    {
      "affs": [
        6
      ],
      "name": "Kim Hoa Nguyen"
    },
    {
      "affs": [
        7
      ],
      "name": "Begali Saytov"
    },
    {
      "affs": [
        0,
        8
      ],
      "name": "Amit Sedai"
    },
    {
      "affs": [
        0,
        8
      ],
      "name": "Kumari Ankita"
    },
    {
      "affs": [
        0,
        8
      ],
      "name": "Rajat Kumar Agarwal"
    },
    {
      "affs": [
        0,
        9
      ],
      "name": "Lawrence Faulkner"
    }
  ],
  "bibtex": "https://research.draconitic.com/paper/dfvp9wnx7bmch.bib",
  "cited_by": 1,
  "doi": "10.1182/bloodadvances.2025019083",
  "fulltext_url": "https://pmc.ncbi.nlm.nih.gov/articles/PMC13737386/",
  "id": "dfvp9wnx7bmch",
  "issns": [
    "2473-9529",
    "2473-9537"
  ],
  "landing_url": "https://doi.org/10.1182/bloodadvances.2025019083",
  "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",
      "Transplantation"
    ],
    [
      "openalex",
      "2720|Hematopoietic Stem Cell Transplantation"
    ]
  ],
  "openalex": "W7169528113",
  "pdf_url": null,
  "pmcid": "PMC13737386",
  "pmid": "42469166",
  "published": "2026-07-22",
  "publisher": "The American Society of Hematology",
  "ris": "https://research.draconitic.com/paper/dfvp9wnx7bmch.ris",
  "sources": [
    {
      "first_seen": "2026-11-09T12:22:35Z",
      "native_id": "PMC13737386",
      "source": "pmc",
      "url": "https://doi.org/10.1182/bloodadvances.2025019083"
    }
  ],
  "subjects": [
    {
      "field": "medicine",
      "origin": "openalex:2720|Hematopoietic Stem Cell Transplantation",
      "subfield": "general"
    }
  ],
  "title": "Optimizing outcomes and accessibility of matched sibling donor transplant for transfusion-dependent thalassemia in LMICs",
  "title_text": "Optimizing outcomes and accessibility of matched sibling donor transplant for transfusion-dependent thalassemia in LMICs",
  "type": "peer-reviewed",
  "updated_at": "2026-11-09T12:22:35Z",
  "url": "https://research.draconitic.com/paper/dfvp9wnx7bmch",
  "venue": "Blood Advances",
  "versions": []
}