SKION

PK/PD of corticosteroids in graft-versus-host                      PIKACHU-study
disease after hematopoietic cell transplantation
in children.

Pediatric subjects with chronic Graft Versus Host                Ibrutinib (PCYC-1146-IM)
Disease.                                                  

Patients less than 18 years of age with histologically             BIANCA
confirmed B-cell NHL who have relapsed after
one or more prior therapies or are primary refractory.  

Pediatric HSCT recipients transplanted from a                       Tracing HSCs after transplantation
sibling donor, with >95% donor chimerism and
without major HSCT-related complications.

Tisagenlecleucel (Kymriah ®) as standard of care                 Tisagenlecleucel

Patients 12-65 years of age with a diagnosis of                      NiCord III       
hematological malignancy who are candidates
for unrelated cord blood transplantation, with 
qualifying HLA-matched UCB units with sufficient 
pre-cryopreserved total nucleated cell dose and 
CD34+ cell dose (see protocol for further specs)

Pediatric patients (0-18 years old) receiving a                        PARACHUTE
non-T-cell depleted allogeneic hematopoietic                        (gesloten voor inclusie)
cell transplantation with Thymoglobulin in the
conditioning regimen as standard of care.   

Lange termijn follow up      

Per Health Authorities guidelines for gene                             PAVO
therapy medicinal products that utilize 
integrating vectors (e.g. lentiviral vectors),
long term safety and efficacy follow-up of
treated patients is required.
The purpose of this study is to monitor all
patients exposed to CAR T-cells (CAR-T)
for 15 years following last CAR-T (e.g.CTL019) 
infusion to assess the risk of delayed
adverse events (AEs) and assess
long term efficacy, including vector 
persistence.