Ef64257; cacy and toxicity of non-T-cell depleted haploidentical stem cell transplantation in children with refractory or relapsed acute leukaemia

Ref ID: 18547


A. Kikuta, H. Sano, S. Kobayashi, M. Akaihata, M. Ito,
K. Mochizuki, H. Ohto

Author address:

Fukushima Medical University (Fukushima, JP)

Full conference title:

Annual Meeting of the EBMT, 38th


Background: Non-T-cell depleted (non-TCD) HLA-haploidentical
SCT (haplo-SCT) is a form of adoptive cellular therapy that
has a high degree of effi cacy in hematologic malignancies.
Previously we reported the safety profi le assessing GVHD
prophylaxis that was conducted with anti-human thymocyte
immunoglobulin(ATG), tacrolimus, methotrexate(MTX) and
prednisolone(PSL) in non-TCD haplo-SCT (Kikuta, Clin Transplant, 2010). We evaluated effi cacy and toxicity of non-TCD
haplo-SCT in children with very high risk refractory/relapsed
acute leukemia (VHR-R/R AL).
Methods: VHR-R/R AL was defi ned by the 1-year survival rate
less than 30%. From Aug 2000 to April 2011, consecutive 16
patients (pts) with VHR-R/R AL who underwent non-TCD-haploSCT were included. The median age of pts was 7.7(0.5-17.9)
years old. The diagnosis included ALL (10), AML (3), M/NKL (3).
The disease status at non-TCD-haplo-SCT were 4 in CR2 (MLL
rearrangement: 1 pt, Ph positive: 1 pt, after SCT: 2 pts), 12 in
non-CR (after SCT: 5 pts, after chemotherapy: 7 pts). HLA disparities were 3/8 in 1 pt, 4/8 in 15 pts. Donors included fathers
(9), mothers (5), and siblings (2). Fifteen pts received myeloablative conditioning (TBI based: 11 pts, Bu based: 4 pts) and
12 pts of them received ATG (rabbit, thymoglobulin 2.5 mg/kg)
containing regimen. The GVHD prophylaxis was conducted S117
with tacrolimus, MTX and PSL. Thirteen pts received peripheral
blood stem cells and 3 pts received BM.
Results: All pts achieved engraftment (median 14 days for
neutro phils). All pts achieved CR in non-remission at SCT.
Acute GVHD grade 2-4 and grade 3-4 occurred in 12/16 pts
(75%) and 2/16 (13%), respectively. Chronic GVHD occurred
in 7/13 (54%). The treatment-related complications observed
within day+100 included: viral reactivations (14 pts), Candida sepsis (2 pts), Aspergillus (1 pt), Bacterial sepsis (2 pts),
hemorrhagic cystitis (2 pts), thrombotic microangiopathy (1 pt),
and posterior reversible encephalopathy syndrome (1 pt). Nonrelapsed mortality occurred in 3 pts and relapse occurred in
3 pts. With the median follow-up of 15 (1.5-134) months, 1-year
and 2-year event free survival were 69% and 59%.
Conclusions: These data suggest that non-TCD haplo-SCT
combined with our GVHD prophylaxis is well tolerated, facilitate engraftment, and has signifi cant anti-tumor activity, particularly in pediatric patients with non-remission acute leukemia.
The safety profi le is acceptable in this refractory/ relapsed

Abstract Number: P457

Conference Year: 2012

Link to conference website: NULL

New link: NULL

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