Successful outcome of allogeneic stem cell transplantation in Seckel syndrome

Pediatr Transplant. 2014 May;18(3):E93-5. doi: 10.1111/petr.12230. Epub 2014 Feb 1.

Abstract

Seckel syndrome is a rare autosomal recessive disease, genetically heterogeneous, characterized by short stature, prenatal microcephaly, intellectual disability, dysmorphic features, chromosomal instability, and hematological disorders. We report the case of a six-yr-old boy with Seckel syndrome and aplastic anemia who underwent successful allogeneic bone marrow transplantation from ten of ten HLA matched unrelated donor. Currently the patient is on D+771, in good health conditions and with no further complications. In conclusion, this case indicates that bone marrow transplantation is an acceptable therapeutic option for Seckel syndrome complicated by hematological alterations.

Keywords: allogeneic stem cell transplantation; bone marrow transplantation; children; pediatrics.

Publication types

  • Case Reports

MeSH terms

  • Alleles
  • Anemia, Aplastic / complications
  • Anemia, Aplastic / therapy*
  • Bone Marrow Transplantation
  • Child
  • Cyclosporine / therapeutic use
  • Dwarfism / complications
  • Dwarfism / therapy*
  • Facies
  • Female
  • HLA Antigens
  • Humans
  • Living Donors
  • Male
  • Microcephaly / complications
  • Microcephaly / therapy*
  • Middle Aged
  • Stem Cell Transplantation / methods*
  • Transplantation Conditioning
  • Transplantation, Homologous / methods
  • Treatment Outcome

Substances

  • HLA Antigens
  • Cyclosporine

Supplementary concepts

  • Seckel syndrome 1