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Conditioning Regimens

Fludarabine-based disease-specific conditioning or conventional myeloablative conditioning in hematopoietic stem cell transplantation for treatment of non-malignant diseases

Bone Marrow Transplantation volume 39, pages 383–388 (2007)Cite this article

Abstract

Fludarabine-based conditioning (FBC) was given to 24 patients and conventional myeloablative conditioning (MC) to 33 patients, most children, before hematopoietic stem cell transplantation (HSCT) for non-malignant diseases. The donors were human leukocyte antigen (HLA)-A, -B, -DRβ1-identical related (33%) or unrelated (67%). In the FBC group, two grafts failed versus three in the MC group; all were successfully regrafted. Fever was more common in the MC patients (P=0.003). Bacteremia occurred in 25% of the FBC group and 50% in the MC group (P=0.1). In the FBC group, platelet engraftment was faster and transfusions were fewer (P<0.05). Mucositis and renal function were similar in the two groups. The MC group had higher maximum bilirubin (P=0.03) and less often normal spirometry (P=0.04) after HSCT. A 7-year-old girl in the MC group had permanent alopecia. No patients had severe acute graft-versus-host disease (GVHD). Chronic GVHD was rare. Complete donor CD3+ chimerism was more common in the MC group (P=0.01), but CD33+ engraftment was better with FBS (P=0.03). Treatment-related mortality was 4 and 15%, and 5-year survival was 89 and 85% in the FBC and MC groups. Although survival was similar, FBC is a promising alternative to MC in non-malignant disorders.

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Acknowledgements

We thank the staff for compassionate and competent patient care. Inger Hammarberg is acknowledged for skilful preparation of this paper. Supported by grants from the Swedish Cancer Society (0070-B05-19XAC), the Children's Cancer Foundation (03/039), the Swedish Research Council (K2006-32X-05971-26-1), the Cancer Society in Stockholm, the Cancer- and Allergy Foundation, and Karolinska Institutet.

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Authors and Affiliations

  1. Division of Clinical Immunology, Karolinska Institutet, Karolinska University Hospital, Stockholm, Huddinge, Sweden

    O Ringdén, M Remberger, M Uzunel, L Barkholt & J Mattsson

  2. Center for Allogeneic Stem Cell Transplantation (CAST), Karolinska Institutet, Karolinska University Hospital, Stockholm, Huddinge, Sweden

    O Ringdén, M Remberger, P Svenberg, B-M Svahn, Z Hassan, B Omazic, J Aschan, L Barkholt & J Mattsson

  3. Department of Pediatric Dentistry, Karolinska Institutet, Karolinska University Hospital, Stockholm, Huddinge, Sweden

    G Dahllöf

  4. Department of Pediatrics, Karolinska Institutet, Karolinska University Hospital, Stockholm, Huddinge, Sweden

    B Gustafsson & J Winiarski

  5. Department of Hematology, Karolinska Institutet, Karolinska University Hospital, Stockholm, Huddinge, Sweden

    J Aschan & P Ljungman

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  1. O Ringdén
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Correspondence to O Ringdén.

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Ringdén, O., Remberger, M., Svenberg, P. et al. Fludarabine-based disease-specific conditioning or conventional myeloablative conditioning in hematopoietic stem cell transplantation for treatment of non-malignant diseases. Bone Marrow Transplant 39, 383–388 (2007). https://doi.org/10.1038/sj.bmt.1705602

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