PMID- 17310137 OWN - NLM STAT- MEDLINE DCOM- 20070516 LR - 20220331 IS - 0268-3369 (Print) IS - 0268-3369 (Linking) VI - 39 IP - 7 DP - 2007 Apr TI - Fludarabine-based disease-specific conditioning or conventional myeloablative conditioning in hematopoietic stem cell transplantation for treatment of non-malignant diseases. PG - 383-8 AB - 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, -DRbeta1-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. FAU - Ringden, O AU - Ringden O AD - Division of Clinical Immunology, Karolinska Institutet, Karolinska University Hospital, Huddinge, Stockholm, Sweden. Olle.Ringden@ki.se FAU - Remberger, M AU - Remberger M FAU - Svenberg, P AU - Svenberg P FAU - Svahn, B-M AU - Svahn BM FAU - Dahllof, G AU - Dahllof G FAU - Gustafsson, B AU - Gustafsson B FAU - Hassan, Z AU - Hassan Z FAU - Omazic, B AU - Omazic B FAU - Uzunel, M AU - Uzunel M FAU - Aschan, J AU - Aschan J FAU - Barkholt, L AU - Barkholt L FAU - Winiarski, J AU - Winiarski J FAU - Ljungman, P AU - Ljungman P FAU - Mattsson, J AU - Mattsson J LA - eng PT - Journal Article PT - Research Support, Non-U.S. Gov't DEP - 20070219 PL - England TA - Bone Marrow Transplant JT - Bone marrow transplantation JID - 8702459 RN - 0 (Antineoplastic Agents) RN - FA2DM6879K (Vidarabine) RN - P2K93U8740 (fludarabine) SB - IM MH - Adolescent MH - Adult MH - Anemia, Aplastic/*therapy MH - Antineoplastic Agents/*pharmacology MH - Child MH - Child, Preschool MH - Female MH - Graft vs Host Disease MH - Hematopoietic Stem Cell Transplantation/*methods MH - Hemoglobinopathies/*therapy MH - Humans MH - Immunologic Deficiency Syndromes/*therapy MH - Infant MH - Male MH - Middle Aged MH - *Transplantation Conditioning MH - Vidarabine/*analogs & derivatives/pharmacology EDAT- 2007/02/21 09:00 MHDA- 2007/05/17 09:00 CRDT- 2007/02/21 09:00 PHST- 2007/02/21 09:00 [pubmed] PHST- 2007/05/17 09:00 [medline] PHST- 2007/02/21 09:00 [entrez] AID - 1705602 [pii] AID - 10.1038/sj.bmt.1705602 [doi] PST - ppublish SO - Bone Marrow Transplant. 2007 Apr;39(7):383-8. doi: 10.1038/sj.bmt.1705602. Epub 2007 Feb 19.