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: 2.35). 04/2012; 59(2):372-6. DOI: 10.1002/pbc.24177
Source: PubMed

ABSTRACT 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: Despite improvements in medical management, sickle cell disease (SCD) remains associated with severe morbidity and decreased survival. Allogeneic haematopoietic stem cell transplantation (HSCT) remains the only curative approach. We report the outcome of 50 consecutive children with severe SCD that received HSCT in our unit between November 1988 and April 2013. The stem cell source was bone marrow (n = 39), cord blood (n = 3), bone marrow and cord blood (n = 7) and peripheral blood stem cells (n = 1). All patients had ≥1 severe manifestation: 37 presented with recurrent vaso-occlusive crises/acute chest syndrome, 27 cerebral vasculopathy and 1 nephropathy. The conditioning regimen consisted of busulfan + cyclophosphamide (BuCy) before November 1991 and BuCy + rabbit antithymocyte globulin after that date. Since 1995, all patients have been treated with hydroxycarbamide (HC) prior to transplantation for a median duration of 2·7 years. Median age at transplantation and median follow-up was 8·3 and 7·7 years, respectively. Acute graft-versus-host disease (GVHD) and chronic GVHD were observed in 11 and 10 patients, respectively. An excellent outcome was achieved, with 8-year overall survival and event-free survival (EFS) rates of 94·1% and 85·6%, respectively. Since HC introduction, no graft failure occurred and EFS reached 97·4%. Prior treatment with HC may have contributed to successful engraftment.
    British Journal of Haematology 01/2014; · 4.94 Impact Factor
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    ABSTRACT: Background Hematopoietic stem cell transplantation (HSCT) is an accepted treatment for pediatric malignant and non-malignant conditions. Agreeing to HSCT can be challenging for families. This study explored themes reported by family members as influencing their acceptance of HSCT as a necessary treatment intervention.ProcedureIn a four-site study, 107 individuals within 30 families pursuing HSCT for a pediatric malignancy, and 25 individuals within 6 families pursuing HSCT for a pediatric non-malignancy were interviewed pre-HSCT. Semantic content analysis was used in this secondary analysis to identify relevant themes.ResultsFifty-eight parents (and surrogates) 19 patients, 16 donor siblings, and 39 non-donor siblings, half-siblings and cousins, participated. Thirteen themes were identified as influencing the acceptance of HSCT. The most frequently reported were: “recommendation by the child's physician,” viewing HSCT as the “best chance for cure,” and “desiring a more normal and better quality of life for the patient and family.” Seven themes were reported by all categories of family members, though at different frequencies. Two themes (“HSCT being part of the upfront treatment plan”: “hearing of HSCT success in others”) were only reported by the malignancy group, and one theme (“worrying about disease progression and losing a window of HSCT opportunity”) was only reported by the non-malignancy group.Conclusion Parents, patients, and other family members can articulate multiple themes that influence their considerations of HSCT. Understanding these themes may guide discussions between families and healthcare teams. Pediatr Blood Cancer © 2014 Wiley Periodicals, Inc.
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