Cord blood transplantation in children with hemoglobinopathies

Luisa Strocchio, Mariateresa Romano, Maria Giuseppina Cefalo, Luciana Vinti, Stefania Gaspari, Franco Locatelli

Research output: Contribution to journalArticlepeer-review


Introduction: Current challenges in the field of hematopoietic stem cell transplantation (HSCT) for hemoglobinopathies include the possibility of using alternative donors to expand the number of candidates to an allograft. Umbilical cord blood transplantation (UCBT) have extended the access to HSCT in patients lacking matched related or unrelated adult donors, and appears particularly appealing in non-malignant diseases by virtue of the associated low incidence of graft-versus-host disease (GVHD).Areas covered: Outcomes of patients with thalassemia or sickle cell disease given related UCBT is at least as good as that of patients receiving bone marrow (BM) allograft from human leucocyte antigen (HLA)-identical siblings, provided the UCB unit contains adequate cell dose. The experience with unrelated UCBT has been less encouraging, primarily due to low engraftment rates and delayed hematopoietic recovery. New promising approaches of ex vivo graft manipulation aimed at overcoming the drawback of cell dose limitation are under investigation.Expert opinion: We suggest that HSCT should be pursued whenever an HLA-identical CB unit is available, employing it either alone or in combination with BM from the same donor. Based on currently reported data, unrelated UCBT appears to be a suboptimal strategy for patients with hemoglobinopathies, unless it is performed in the context of clinical trials aimed at exploring specific treatment platforms of ex vivo UCB-graft manipulation.

Original languageEnglish
Pages (from-to)1125-1136
Number of pages12
JournalExpert Opinion on Orphan Drugs
Issue number10
Publication statusPublished - Oct 3 2015


  • cord blood transplantation
  • hemoglobinopathies
  • sickle cell disease
  • thalassemia

ASJC Scopus subject areas

  • Pharmacology (medical)
  • Health Policy
  • Pharmacology, Toxicology and Pharmaceutics (miscellaneous)


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