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Table 1 Transplant-related data of patients

From: Farber disease: clinical presentation, pathogenesis and a new approach to treatment

Parameter Patients
Patient number and gender #1, female #2, male #3, female #4, female
Patient origin Muenster Muenster Muenster Hamburg
Age at SCT 3 years 3 years 2 years 21 years
Preparative regimen Bu/Cy Bu/Cy/ATG Bu/Cy Bu/Cy/ATG
Stem cell source and dose (no T-cell depletion) BM (MRD), 11.3 × 106 CD34 PBSC (MUD), 25 × 106 CD34 BM (MRD), 5.6 × 106 CD34 BM (MRD), 4.2 × 106 CD34
GvHD prophylaxis CsA, MTX CsA, MTX CsA, MTX CsA, MTX
Neutrophil engraftment day +14 day +12 day +18 day +14
Acute/chronic GvHD grade II of gut/none grade I of gut/none grade II of skin/none none
Therapy of acute/chronic GvHD steroids/none steroids/none steroids + tacrolimus/none none
Infections Clostridium difficile enteritis, bacteremia with Staphylococci CMV reactivation CMV colitis none
Toxicities CsA-neurotoxicity None CsA-neurotoxicity suspected VOD
Current donor cell chimerism 91% 95% 90% 99%