Role of Pharmacogenetics in Hematopoietic Stem Cell Transplantation Outcome in Children.
Hematopoietic stem cell transplantation (HSCT) is an established therapeutic procedure for several congenital and acquired disorders, both malignant and nonmalignant. Despite the great improvements in HSCT clinical practices over the last few decades, complications, such as graft vs. host disease (GVHD) and sinusoidal obstructive syndrome (SOS), are still largely unpredictable and remain the major causes of morbidity and mortality. Both donor and patient genetic background might influence the success of bone marrow transplantation and could at least partially explain the inter-individual variability in HSCT outcome. This review summarizes some of the recent studies on candidate gene polymorphisms in HSCT, with particular reference to pediatric cohorts. The interest is especially focused on pharmacogenetic variants affecting myeloablative and immunosuppressive drugs, although genetic traits involved in SOS susceptibility and transplant-related mortality are also reviewed.
Institute for Maternal and Child Health (I.R.C.C.S.) Burlo Garofolo, UO Pediatric Hemato-Oncology, Trieste 34137, Italy. rfranca@units.it.
Raffaella
R
Franca
Department of Life Sciences, University of Trieste, Trieste 34127, Italy. stoccog@units.it.
Gabriele
G
Stocco
Institute for Maternal and Child Health (I.R.C.C.S.) Burlo Garofolo, UO Pediatric Hemato-Oncology, Trieste 34137, Italy. dieg.o@libero.it.
Diego
D
Favretto
Institute for Maternal and Child Health (I.R.C.C.S.) Burlo Garofolo, UO Pediatric Hemato-Oncology, Trieste 34137, Italy. nagua.giurici@burlo.trieste.it.
Nagua
N
Giurici
Department of Life Sciences, University of Trieste, Trieste 34127, Italy. decorti@units.it.
Giuliana
G
Decorti
Institute for Maternal and Child Health (I.R.C.C.S.) Burlo Garofolo, UO Pediatric Hemato-Oncology, Trieste 34137, Italy. marco.rabusin@burlo.trieste.it.
Marco
M
Rabusin
D002648:Child; D006086:Graft vs Host Disease / Q000188:drug therapy / Q000453:epidemiology / Q000235:genetics*; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods*; D006504:Hepatic Veno-Occlusive Disease / Q000188:drug therapy / Q000453:epidemiology / Q000235:genetics*; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D010597:Pharmacogenetics / Q000379:methods*; D011110:Polymorphism, Genetic; D016896:Treatment Outcome
D007166:Immunosuppressive Agents
graft vs. host disease; hematopoietic stem cell transplantation; pharmacogenetics; sinusoidal obstructive syndrome