[Single institution experience of HLA-haploidentical hematopoietic stem cell transplantation for children: current problems and perspective]

Rinsho Ketsueki. 2014 Jan;55(1):110-9.
[Article in Japanese]

Abstract

HLA-haploidentical 2 or 3-loci mismatched families are alternative donors for high-risk patients without HLA-matched donors. We retrospectively reviewed our case series of HLA-halpoidentical hematopoietic stem cell transplantations (haplo-HSCTs). Between Jul 2005 and Dec 2012, 25 patients (median age, 8 y; 13 ALL, 8 AML, 4 others) received haplo-HSCTs because of a worsening prognosis (i.e. induction failure, non-CR, or relapse after prior HSCT). Disease status was CR in 8 and non-CR in 17 patients. The 17 patients received myeloablative conditioning, while the 8 were given reduced-intensity conditioning because of their conditions (e.g. early relapse after prior HSCT). ATG was not administered in all but 3 patients. Tacrolimus and sMTX were used for prophylaxis GVHD and steroids were immediately given to prevent the onset of aGVHD. The 3-year OS and EFS were 35.6±10.0% and 31.3±10.1%, respectively (median follow-up, 49 mo); 14 patients died of their primary disease. Grade 3-4 aGVHD occurred in 7 patients, 2 of whom died of grade 4 aGVHD. Eleven patients had extensive cGVHD. While 4 of the 8 CR patients remained in CR, only 4 of the 17 non-CR patients achieved long-term CR (survival time, 6-89 mo). Haplo-HSCT was tolerable with strict control of infections and GVHD. However, further strategies for non-CR patients appear to be required.

Publication types

  • English Abstract

MeSH terms

  • Adolescent
  • Child
  • Child, Preschool
  • Female
  • Graft vs Host Disease / prevention & control
  • HLA Antigens / genetics*
  • Haploidy*
  • Hematopoietic Stem Cell Transplantation* / mortality
  • Histocompatibility / genetics*
  • Humans
  • Immunosuppressive Agents / administration & dosage
  • Infant
  • Leukemia, Myeloid, Acute / mortality
  • Leukemia, Myeloid, Acute / therapy*
  • Male
  • Methotrexate / administration & dosage
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma / mortality
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma / therapy*
  • Remission Induction
  • Retrospective Studies
  • Survival Rate
  • Tacrolimus / administration & dosage
  • Transplantation Conditioning

Substances

  • HLA Antigens
  • Immunosuppressive Agents
  • Tacrolimus
  • Methotrexate