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Low Exposure Busulfan Conditioning to Achieve Sufficient Multilineage Chimerism in Patients with Severe Combined Immunodeficiency.
Dvorak, Christopher C; Long-Boyle, Janel; Dara, Jasmeen; Melton, Alexis; Shimano, Kristin A; Huang, James N; Puck, Jennifer M; Dorsey, Morna J; Facchino, Janelle; Chang, Catherine K; Cowan, Morton J.
Afiliação
  • Dvorak CC; Division of Pediatric Allergy, Immunology, and Bone Marrow Transplantation, University of California San Francisco Benioff Children's Hospital, San Francisco, California. Electronic address: dvorakc@peds.ucsf.edu.
  • Long-Boyle J; Division of Pediatric Allergy, Immunology, and Bone Marrow Transplantation, University of California San Francisco Benioff Children's Hospital, San Francisco, California; Department of Clinical Pharmacy, University of California San Francisco, San Francisco, California.
  • Dara J; Division of Pediatric Allergy, Immunology, and Bone Marrow Transplantation, University of California San Francisco Benioff Children's Hospital, San Francisco, California.
  • Melton A; Division of Pediatric Allergy, Immunology, and Bone Marrow Transplantation, University of California San Francisco Benioff Children's Hospital, San Francisco, California.
  • Shimano KA; Division of Pediatric Allergy, Immunology, and Bone Marrow Transplantation, University of California San Francisco Benioff Children's Hospital, San Francisco, California; Division of Pediatric Hematology and Oncology, University of California San Francisco Benioff Children's Hospital, San Francisco,
  • Huang JN; Division of Pediatric Allergy, Immunology, and Bone Marrow Transplantation, University of California San Francisco Benioff Children's Hospital, San Francisco, California; Division of Pediatric Hematology and Oncology, University of California San Francisco Benioff Children's Hospital, San Francisco,
  • Puck JM; Division of Pediatric Allergy, Immunology, and Bone Marrow Transplantation, University of California San Francisco Benioff Children's Hospital, San Francisco, California.
  • Dorsey MJ; Division of Pediatric Allergy, Immunology, and Bone Marrow Transplantation, University of California San Francisco Benioff Children's Hospital, San Francisco, California.
  • Facchino J; Division of Pediatric Allergy, Immunology, and Bone Marrow Transplantation, University of California San Francisco Benioff Children's Hospital, San Francisco, California.
  • Chang CK; Division of Pediatric Allergy, Immunology, and Bone Marrow Transplantation, University of California San Francisco Benioff Children's Hospital, San Francisco, California.
  • Cowan MJ; Division of Pediatric Allergy, Immunology, and Bone Marrow Transplantation, University of California San Francisco Benioff Children's Hospital, San Francisco, California.
Biol Blood Marrow Transplant ; 25(7): 1355-1362, 2019 07.
Article em En | MEDLINE | ID: mdl-30876930
After allogeneic hematopoietic cell transplantation (HCT), the minimal myeloid chimerism required for full T and B cell reconstitution in patients with severe combined immunodeficiency (SCID) is unknown. We retrospectively reviewed our experience with low-exposure busulfan (cumulative area under the curve, 30 mg·hr/L) in 10 SCID patients undergoing either first or repeat HCT from unrelated or haploidentical donors. The median busulfan dose required to achieve this exposure was 5.9 mg/kg (range, 4.8 to 9.1). With a median follow-up of 4.5 years all patients survived, with 1 requiring an additional HCT. Donor myeloid chimerism was generally >90% at 1 month post-HCT, but in most patients it fell during the next 3 months, such that 1-year median myeloid chimerism was 14% (range, 2% to 100%). Six of 10 patients had full T and B cell reconstitution, despite myeloid chimerism as low as 3%. Three patients have not recovered B cell function at over 2 years post-HCT, 2 of them in the setting of treatment with rituximab for post-HCT autoimmunity. Low-exposure busulfan was well tolerated and achieved sufficient myeloid chimerism for full immune reconstitution in over 50% of patients. However, other factors beyond busulfan exposure may also play critical roles in determining long-term myeloid chimerism and full T and B cell reconstitution.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Bussulfano / Linfócitos B / Linfócitos T / Imunodeficiência Combinada Severa / Quimeras de Transplante / Condicionamento Pré-Transplante Tipo de estudo: Observational_studies / Prognostic_studies Limite: Child / Female / Humans / Infant / Male Idioma: En Ano de publicação: 2019 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Bussulfano / Linfócitos B / Linfócitos T / Imunodeficiência Combinada Severa / Quimeras de Transplante / Condicionamento Pré-Transplante Tipo de estudo: Observational_studies / Prognostic_studies Limite: Child / Female / Humans / Infant / Male Idioma: En Ano de publicação: 2019 Tipo de documento: Article