14800-DvRappard

78 Chapter 5 patients (36%) died 22 to 72months after diagnosis. Overall survival at latest assessment was 76.9% for transplanted and 63.6% for non-transplanted patients (Figure 1B). One patient experienced acute, 3 chronic graft-versus-host disease (1 extensive). All were effectively successfully treated with corticosteroids and came off immunosuppressive therapy. Figure 1 : Patient cohort and outcome after HCT. (A) 13 transplanted patients (magenta shades, 6 asymptomatic (diagnosed because of an affected sibling), mean age 14.4 years, range 2-35 years) and 22 non-transplanted patients (blue shades, mean age 6.5 years, range 2-32 years). Four patients were referred fromother European countries (Belgium, Denmark and Luxemburg); the remainder came from the Netherlands. (B) Overall survival probability for transplanted and non-transplanted patients. (C) Probability of IFS and AFS for transplanted and non-transplanted patients. (D) Probability of IFS and AFS for symptomatic (n=7) and presymptomatic (n=6) transplanted patients. IFS (whereby death, wheelchair dependency, gastrostomy and intrathecal baclofen treatment were regarded as events) was 69.2% for transplanted and 9.1% for non- transplanted patients ( P =.03; Figure 1C). Symptomatic transplanted patients had lower estimated IFS (42.9%) than presymptomatic transplanted patients (100%) at HCT ( P =.052; Figure 1D). AFS was defined as no occurrence of death, motor (clinically relevant peripheral neuropathy, spasticity or ataxia, gross motor function ≥ 3) or cognitive (IQ decline ≥ 6 points) deterioration. Transplanted patients had higher AFS (46.2%) than nontransplantedpatients (0%; P =.01; Figure 1C). Symptomatic transplantedpatients had