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Targeted busulfan-based conditioning unrelated donor bone marrow transplantation for Diamond-Blackfan anemia
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  • Katsuhide Eguchi,
  • Masataka Ishimura,
  • Akihiro Tamura,
  • Nobuhiro Ito,
  • Naoki Hirano,
  • Shunichi Adachi,
  • Keishiro Kinoshita,
  • Hirokazu Kobushi,
  • Yutaro Yada,
  • Motoshi Sonoda,
  • Akira Shiraishi,
  • Yoshiyuki Kosaka,
  • Kiminori Terui,
  • Etsuro Ito,
  • Shouichi Ohga
Katsuhide Eguchi
Graduate School of Medicial Sciences, Kyushu University
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Masataka Ishimura
Graduate School of Medicial Sciences, Kyushu University

Corresponding Author:[email protected]

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Akihiro Tamura
Kobe Children’s Hospital
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Nobuhiro Ito
Nagasaki University Hospital
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Naoki Hirano
Oita University Faculty of Medicine
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Shunichi Adachi
Graduate School of Medicial Sciences, Kyushu University
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Keishiro Kinoshita
Graduate School of Medicial Sciences, Kyushu University
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Hirokazu Kobushi
Graduate School of Medicial Sciences, Kyushu University
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Yutaro Yada
Graduate School of Medicial Sciences, Kyushu University
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Motoshi Sonoda
Graduate School of Medicial Sciences, Kyushu University
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Akira Shiraishi
Graduate School of Medicial Sciences, Kyushu University
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Yoshiyuki Kosaka
Kobe Children’s Hospital
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Kiminori Terui
Hirosaki University Graduate School of Medicine
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Etsuro Ito
Hirosaki University Graduate School of Medicine
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Shouichi Ohga
Graduate School of Medicial Sciences, Kyushu University
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Abstract

Hematopoietic cell transplantation corrects a hematological phenotype of Diamond-Blackfan anemia (DBA). Reduced conditioning has been recommended for patients with the cancer predisposition, but appropriate intensity is required to prevent graft failure. We conducted unrelated-donor bone marrow transplantations in six consecutive transfusion-dependent patients (median age, 5.4 years; range, 1.8–23) after targeted-busulfan (60–75 mg/L×h of cumulative AUC) and fludarabine regimen without irradiation. All obtained chronic graft-versus-host-disease-free complete donor chimerism, including one unrelated cord blood transplantation rescue. The rescued 7-year-old girl had the longest transfusion-dependency (>40 months) and highest ferritin level (>2,000 ng/mL) pretransplant. Intermediate-intensity targeted busulfan-based conditioning may cure DBA.