Details

Autor(en) / Beteiligte

• Ehlert, K
• Roth, J
• Frosch, M
• Fehse, N
• Zander, N
• Vormoor, J

Titel

Farber’s disease without central nervous system involvement: bone-marrow transplantation provides a promising new approach

Ist Teil von

• Annals of the rheumatic diseases, 2006-12, Vol.65 (12), p.1665-1666

Ort / Verlag

London: BMJ Publishing Group Ltd and European League Against Rheumatism

Erscheinungsjahr

2006

Link zum Volltext

Quelle

BMJ Journals Archiv - DFG Nationallizenzen

Beschreibungen/Notizen

• Table 1 Transplant-related data Patient 1, female (MS) 2, male (MS) 3, female (MS) 4, female (HH) Age at Tx 3 years, 11 months 3 years, 10 months 2 years 21 years Preparative regimen, cumulative dose in mg/kg bw Bu 12.8 iv/Cy 120 iv Bu 12.8 iv/Cy 120 iv/ATG 40 iv Bu 12.8 iv/Cy 120 iv Bu 12 po/Cy 120 iv/ATG 90 iv Donor-stem cell source, dose/kg bw MRD-BM, 11.3x106 CD34 MUD-PBSC, 25x106 CD34 MRD-BM, 5.6x106 CD34 MRD-BM, 4.2x106 CD34 GvHD prophylaxis CsA, MTX CsA, MTX CsA, MTX CsA, MTX Day of neutrophil engraftment 14 12 18 14 Acute/chronic GvHD Grade II of GIT/none Grade I of GIT/none Grade II of skin/none None Treatment of acute GvHD Steroids Steroids Steroids and tacrolimus None Infections Clostridium difficile enteritis, bacteraemia with staphylococci CMV reactivation CMV colitis None Toxicities CsA neurotoxicity None CsA-neurotoxicity Suspected VOD Chimerism (%) 91 96 90 99 Time point of follow-up Before Tx Day 450 Before Tx Day 200 Before Tx Day 270 Before Tx Day 958 ESR (1 h) 38 9 53 12 25 13 No data available Number of nodules 58 8 39 14 18 0 Pre-Tx nodules mainly dissolved, no new nodules Joints with restricted mobility 26 2 24 4 10 4 Almost all joints affected; improvement after HSCT ATG, anti-thymocyte globulin; BM, bone marrow; Bu, busulfan; bw, body weight; CsA, ciclosporin A; Cy, cyclophosphamide; ESR, erythrocyte sedimentation rate; GIT, gastrointestinal tract; GvHD, graft-versus-host disease; HH, Hamburg; iv, intravenous; MRD, matched related donor; MS, Muenster; MTX, methotrexate; MUD, matched unrelated donor; PBSC, peripheral blood stem cells; Tx, transplantation; VOD, veno-occlusive disease.