Allogeneic hematopoietic stem cell transplantation in children with sickle cell disease
Dipartimento di Ematologia ed Oncologia Pediatrica, IRCCS Ospedale Pediatrico Bambino Gesù, Roma, Italy.Pediatric Blood & Cancer (Impact Factor: 2.39). 08/2012; 59(2):372-6. DOI: 10.1002/pbc.24177
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) represents the only curative treatment for sickle cell disease (SCD), being successful in around 85-90% of patients. Mortality and long-term morbidity (including infertility, gonadal failure, and chronic graft-vs.-host disease) associated with conventional approaches curtail the number of patients who undergo allo-HSCT. Recently, it has been demonstrated that cord blood is as effective as and possibly safer than bone marrow in pediatric patients with SCD. Likewise, transplant strategies based on the use of reduced-intensity regimens and the induction of mixed chimerism have been explored to decrease allo-HSCT short- and long-term complications.
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ABSTRACT: Sickle cell disease (SCD) is still associated with substantial morbidity and reduced life expectancy. Disease related mortality rises to 14% in adolescents and young adults. Overall and disease free survival following haematopoietic stem cell transplantation (HSCT) is 90% and 95% respectively. In order to reduce transplant associated late effects, the feasibility of a highly immunosuppressive reduced intensity conditioning regimen was explored in children with SCD and a matched sibling donor. Eight patients (median age 9 years) and symptomatic SCD were included. The conditioning regimen consisted of fludarabine, melphalan and either thiotepa or total lymphoid irradiation plus ATG or alemtuzumab. The graft was bone marrow in 7 and cord blood in one case. The conditioning regimen was well tolerated and no severe infectious complications occurred. All patients displayed mixed chimaerism on day +28. After a median follow-up of four years 3/8 patients have mixed leukocyte chimaerism and 8/8 patients have 100% donor erythropoiesis. HSCT from matched sibling donors following a reduced intensity conditioning regimen was well tolerated and resulted in cure in all patients studied. If confirmed in larger patient cohorts, these observations will have important implications for the indications of HSCT in children with SCD. © 2013 John Wiley & Sons A/S.
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ABSTRACT: Background Allogeneic hematopoietic stem cell transplantation (HSCT) is the only available cure for sickle cell anemia (SCA). HSCT-associated risks are one of many barriers to its widespread use for SCA. The study objective was to assess parent and patient perceptions of HSCT in the era of more widespread knowledge about HSCT for SCA.ProcedureWe surveyed parents of children with SCA and adolescents with SCA using the standard reference gamble paradigm. Survey responses between Africans (A) and African Americans (AA) and between parents and adolescents were compared.ResultsSeventy-two percent (64/89) of the respondents were willing to accept ≥5% risk of mortality, while 57% said they would accept a risk of ≥10% of graft versus host disease (GVHD). Twenty-two percent were unwilling to accept any risk of mortality or GVHD. Risk averseness did not differ between A or AA respondents. Fifty-six percent of respondents were willing to accept infertility post-HSCT.Conclusions These data suggest that the majority of parents and adolescents are willing to accept the current risks associated with matched sibling HSCT for SCA. However, there continue to be significant numbers of parents and adolescents who are unwilling to accept any risk of HSCT-associated mortality or GVHD. Pediatr Blood Cancer © 2015 Wiley Periodicals, Inc.
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