Preclinical modeling of hematopoietic stem cell transplantation - Advantages and limitations

Jessica L. Stolfi, Chien Chun S Pai, William J Murphy

Research output: Contribution to journalReview articlepeer-review

11 Scopus citations


Hematopoietic stem cell transplantation, which was first successfully performed in the 1950s, remains a critical therapeutic modality for treatment of a diverse array of diseases, including a multitude of hematological malignancies, autoimmune disorders, amyloidosis and inherited genetic hematological disorders. Although great advances have been made in understanding and application of this therapy, significant complications still exist, warranting further investigation. Of critical importance, graft-versus-host disease (GVHD), in both acute and chronic forms, remains a major complication of hematopoietic stem cell transplantation, responsible for both the development of chronic illness and morbidity, as well as mortality. Use of an appropriate preclinical model may provide significant insight into the mechanistic pathways leading to the development and progression of graft-versus-host disease, as well as cancer in general. However, existing preclinical modeling systems exhibit significant limitations, and development of models that recapitulate the complex and comprehensive clinical scenario and provide a tool by which therapeutic intervention may be developed and assessed is of utmost importance. Here, we review the present status of the field of graft-versus-host disease research. We discuss and summarize the preclinical models currently in use, as well as their advantages and limitations. Hematopoietic stem cell transplantation (HSCT) remains a critical therapeutic modality for a diverse array of diseases. Although critical advances have been made with this therapy, complications still exist. Application of the appropriate preclinical model can provide insight into pathways leading to successful engraftment versus complication. Here, we summarize HSCT preclinical models currently used and their advantages and limitations.

Original languageEnglish (US)
Pages (from-to)1595-1606
Number of pages12
JournalFEBS Journal
Issue number9
StatePublished - May 1 2016


  • adoptive therapy
  • cancer
  • canine
  • conditioning
  • graft versus tumor
  • graft versus-host disease
  • hematopoietic stem cell transplantation
  • immunotherapy
  • preclinical
  • primate

ASJC Scopus subject areas

  • Biochemistry
  • Cell Biology
  • Molecular Biology


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