P. Sedlacek et al., Hematopoietic stem cell transplantation in children with hematological malignancies across HLA barriers - reasonable alternative?, NEOPLASMA, 48(4), 2001, pp. 302-306
The aim of this study was to evaluate the efficiency and risks of T-cell de
pletion in prevention of graft versus host disease (GVHD) using HLA haploid
entical family donors as an alternative source of hematopoietic stem cells
(HSC) in children with hematological malignancies without suitable matched
donor.
Ten children, median age 12 years (range, 3-17), were transplanted from hap
loidentical family donors for acute lymphoblastic leukemia (n = 4), acute m
yelogenous leukemia (n=2), chronic myelogenous leukemia (n = 2), non-Hodgki
n lymphoma (n = 1) and myelodysplastic syndrome (n = 1). Parents were donor
s for nine, sibling for one patient. T-cell depletion of HSC was performed
using CellPro followed by antiCD2/CD3 depletion in 7, and CliniMacs magneti
c sorting in 3 grafts.
Primary engraftment was achieved in nine patients. Patient with graft failu
re was successfully re-grafted. Primary acute GVHD was diagnosed in one pat
ient who got higher amount of T-cells in the graft. Secondary GVHD was indu
ced by add-backs of lymphocytes in four patients. Three patients developed
chronic GVHD. Four patients died due to transplant related mortality (40%),
one from veno-occlusive disease, two due to CMV pneumonia and one of asper
gillosis with extensive chronic GVHD. Four patients relapsed with leukemia
within 35-98 days post transplant, three without previous signs of GVHD, an
d all died. Two patients are alive and well 26 and 42 months after transpla
nt.
Haploidentical family donors appear to be a reasonable alternative option f
or patients with urgent indications for allogeneic transplant and/or withou
t a matched donor.