Allogeneic hematopoietic stem cell transplantation in children with sickle cell disease.
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.
- Transfusion Medicine 03/2014; · 1.26 Impact Factor
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ABSTRACT: Following hematopoietic SCT (HSCT) for non-malignant disorders (NMDs) variable donor chimerism among lympho-hematopoietic lines may be observed. We retrospectively evaluated early post-HSCT, lineage-sorted (CD3+ and CD15+) peripheral blood leukocyte chimerism data to characterize patterns and assess for association with long-term CD15+ engraftment. 'Early' was defined as the first value obtained between days +14 and +42, 'late' as the last recorded value after day +90. 'High' donor chimerism was defined as 80% on either fraction at all time-points. Patients were classified into four subgroups with respect to early CD3+/CD15+ chimerism patterns (high/low) then analyzed for long-term CD15+ chimerism status. A total of 135 transplants were evaluable, with all three time-points available in 97. Underlying disease, graft source, patient age and conditioning intensity varied. 'Split' early chimerism (discordant high/low CD3+/CD15+ status) was common. Multivariable analysis revealed strong association between conditioning regimen and primary disease on early CD3+/CD15+ chimerism patterns and a dominant predictive effect of early CD15+ chimerism on long-term CD15+ donor engraftment (observed at median day +365). These data may guide real-time clinician decisions (restraint vs intervention, when available) when faced with unfavorable or unusual early lympho-hematopoietic chimerism patterns following HSCT for NMD.Bone Marrow Transplantation advance online publication, 13 January 2014; doi:10.1038/bmt.2013.207.Bone marrow transplantation 01/2014; · 3.00 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.Pediatric Blood & Cancer 05/2014; · 2.35 Impact Factor