{
  "abstract": "Introduction: Sickle cell disease (SCD) affects an estimated 60,000 to 100,000 individuals in Brazil. Despite advances in medical therapies, patients continue to experience complications and reduced life expectancy, particularly in low- and middle-income countries. Allogeneic hematopoietic stem cell transplantation (HSCT) remains the only established curative treatment. Methods: We conducted a retrospective, single-center observational cohort study including all pediatric allogeneic HSCTs performed for sickle cell disease at our institution between 2021 and the present. Results: Since 2021, 18 pediatric patients with SCD underwent HSCT. Ten were male and eight female, with a median age of 8 years (range, 3–18 years). Eleven patients received grafts from haploidentical HLA donors, and seven from HLA-matched sibling donors. Fresh bone marrow was the stem cell source for all transplants. Haploidentical HSCT (n = 11) was performed using fludarabine/cyclophosphamide/thiotepa/total body irradiation (TBI)/anti-thymocyte globulin (ATG), with cyclophosphamide and TBI dose escalation after the first four transplants. HLA-matched sibling donor HSCT (n = 7) used busulfan/cyclophosphamide/ATG. GVHD prophylaxis in haploidentical transplants consisted of post-transplant cyclophosphamide (PTCy) combined with mycophenolate mofetil and either sirolimus (n = 3) or tacrolimus (n = 8). Sibling donor transplants received cyclosporine and methotrexate (n = 7). The mean TNC and CD34+ cell doses were 9.06 × 10⁸/kg (range, 4.97–14.28) and 15 × 10⁶/kg (range, 7.06–28.41), respectively, in the haploidentical group, and 4.80 × 10⁸/kg (range, 1.75–6.98), and 8.51 × 10⁶/kg (range, 3.4–18.12), respectively, in the sibling donor group. Two patients receiving sibling donor grafts also received complementary umbilical cord blood infusions. The median time to neutrophil engraftment was 18.5 days (range, 14–22 days), with a median of 17 days (range, 14–21 days) in the HLA-matched sibling donor group compared with 20 days (range, 14–22 days) in the haploidentical group. The median time to platelet engraftment was 25 days (range, 15–42 days), with a median of 24.5 days (range, 15–30 days) in the haploidentical group and 25 days (range, 15–42 days) in the sibling donor group. One patient did not achieve platelet engraftment. Peripheral blood chimerism was assessed on day +30. Complete donor chimerism was observed in 15 patients, mixed chimerism (94%) in two patients and one patient had 9% donor chimerism following haploidentical HSCT. By day +100, four patients exhibited mixed chimerism (14%–79%), while one patient had not yet undergone evaluation. During follow-up, two patients experienced graft failure with autologous recovery, one primary and one secondary. CMV reactivation occurred in 14 patients, all treated preemptively. Acute GVHD developed in eight patients, involving the skin (grade I, n = 5; grade II, n = 2) and the gastrointestinal tract (grade IV, n = 1). No cases of chronic GVHD were observed. The median follow-up was 719 days (range, 48–1,869 days). Two patients died of sepsis during long-term follow-up. Conclusion: HSCT for SCD is feasible in a resource-limited setting, with favorable outcomes. The incorporation of haploidentical donors has expanded access to curative therapy for patients lacking HLA-matched sibling donors. Despite the small sample size, this experience demonstrates that HSCT for SCD can be safely performed in a tertiary care center.",
  "abstract_shown": true,
  "affiliations": [
    "Beneficência Portuguesa de Sao Paulo, São Paulo, SP, Brazil"
  ],
  "article_type": null,
  "arxiv": null,
  "author_total": 9,
  "authors": [
    {
      "affs": [
        0
      ],
      "name": "Larissa Silva VALADARES"
    },
    {
      "affs": [
        0
      ],
      "name": "Tomas Marzagao BARBUTO"
    },
    {
      "affs": [
        0
      ],
      "name": "Maria Lucia De Martino LEE"
    },
    {
      "affs": [
        0
      ],
      "name": "Fabricio Tera ROMAGNOL"
    },
    {
      "affs": [
        0
      ],
      "name": "Fernanda Cristina MARINHO MANGA"
    },
    {
      "affs": [
        0
      ],
      "name": "Marina Foltran CELA"
    },
    {
      "affs": [
        0
      ],
      "name": "Adriana Maria PAIXãO SOUZA SILVA"
    },
    {
      "affs": [
        0
      ],
      "name": "Phillip Victor SCHEINBERG"
    },
    {
      "affs": [
        0
      ],
      "name": "Victor Gottardello ZECCHIN"
    }
  ],
  "bibtex": "https://research.draconitic.com/paper/509sv68rpp810.bib",
  "cited_by": 0,
  "doi": "10.1016/j.htct.2026.106896",
  "fulltext_url": "http://www.sciencedirect.com/science/article/pii/S2531137926006437",
  "id": "509sv68rpp810",
  "issns": [
    "2531-1379",
    "2531-1387"
  ],
  "landing_url": "https://doi.org/10.1016/j.htct.2026.106896",
  "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",
      "2716|Hemoglobinopathies and Related Disorders"
    ]
  ],
  "openalex": "W7223101847",
  "pdf_url": null,
  "pmcid": null,
  "pmid": null,
  "published": "2026-10-23",
  "publisher": "Elsevier",
  "ris": "https://research.draconitic.com/paper/509sv68rpp810.ris",
  "sources": [
    {
      "first_seen": "2026-10-30T13:43:22Z",
      "native_id": "f5934e0ffd474db2a8ffe59a300d49e3",
      "source": "doaj",
      "url": "https://doi.org/10.1016/j.htct.2026.106896"
    }
  ],
  "subjects": [
    {
      "field": "medicine",
      "origin": "openalex:2716|Hemoglobinopathies and Related Disorders",
      "subfield": "human-genetics"
    }
  ],
  "title": "PEDIATRIC HEMATOPOIETIC STEM CELL TRANSPLANTATION IN SICKLE CELL DISEASE, EXPERIENCE OF A BRAZILIAN PRIVATE SERVICE",
  "title_text": "PEDIATRIC HEMATOPOIETIC STEM CELL TRANSPLANTATION IN SICKLE CELL DISEASE, EXPERIENCE OF A BRAZILIAN PRIVATE SERVICE",
  "type": "peer-reviewed",
  "updated_at": "2026-10-30T13:43:22Z",
  "url": "https://research.draconitic.com/paper/509sv68rpp810",
  "venue": "Hematology, Transfusion and Cell Therapy",
  "versions": []
}