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Published on: May 7, 2012
Pediatric immunohematopoietic stem cell transplantation at a tertiary care center in Cape Town
Lucille Wood1, June Juritz, Jonathan Havemann
1Department of Haematology, Constantiaberg Medi-Clinic, Plumstead, Cape Town, South Africa.
Insights
This study found that age, diagnosis, graft type, and gender did not impact survival rates in pediatric patients undergoing hematopoietic stem cell transplantation. Standardized regimens are effective for children, but high-risk procedures require accredited teams, especially in developing countries.
Area of Science:
- Pediatric Hematology
- Immunology
- Transplantation Science
Background:
- Pediatric hematopoietic stem cell transplantation (HSCT) is a critical treatment for various hematologic malignancies and non-malignant disorders.
- Assessing factors influencing survival in pediatric HSCT is crucial for optimizing treatment protocols and resource allocation.
- Previous studies have focused on adult populations, necessitating specific analysis for pediatric cohorts.
Purpose of the Study:
- To retrospectively analyze survival outcomes in pediatric patients (<18 years) undergoing HSCT.
- To determine the influence of age, diagnosis, graft type, and gender on survival.
- To compare program activity and outcomes with a state hospital and evaluate program effectiveness.
Main Methods:
- Retrospective analysis of 68 consecutive HSCT procedures in 61 pediatric patients over 6 years.
- Standardized conditioning regimens involving irradiation and cyclophosphamide or busulfan, with T-cell depletion for allografts using Campath 1H.
- Ex vivo T-cell depletion for allografts and no additional immunosuppression unless using matched unrelated donors.
Main Results:
- Survival rates were not significantly affected by age, diagnosis, graft type, or gender in the studied pediatric population.
- Favorable outcomes were observed in patients with aplasia, thalassemia, and sickle cell disease.
- Outcomes for acute myeloid leukemia and hematologic malignancy subgroups showed high survival rates, while acute lymphoblastic leukemia had a higher mortality.
Conclusions:
- Standardized preparative and immunosuppressive regimens are effective for pediatric HSCT, comparable to adult outcomes.
- In resource-limited settings, high-risk, expensive HSCT procedures should be restricted to accredited, active teams in both public and private sectors.
- The findings underscore the importance of specialized teams and accreditation for complex procedures in developing countries.
Unlabelled:
INTRODUCTION AND STUDY DESIGN: We conducted a retrospective analysis of consecutive referrals of patients under 18 years of age undergoing immunohematopoietic stem cell transplantation to assess the influence of age, diagnosis, graft type and gender on survival. We also contrasted program activity and outcome to that reported from a state hospital in the same geographical area over a comparable period.
Methods:
Conditioning employed either a sequential combination of fractionated 12Gy whole body and 6Gy total nodal irradiation separated by 120mg/kg of cyclophosphamide in patients over 15 years of age. Alternatively, the latter agent was combined initially with oral busulphan and later the intravenous equivalent. Neuroblastoma cases were prepared using a different regimen. In allografts the harvested product underwent ex vivo T-cell depletion with the humanized version of anti-CD 52 monoclonal antibody designated Campath 1H. No additional immunosuppression was given except where matched unrelated volunteer donors were employed.
Results:
Sixty-eight procedures were carried out in 61 patients over a 6-year period. Of 11 with acute myeloid leukemia, 8 are alive and well whereas 8 of the 14 with the lymphoblastic variant have died. Of the remaining 12 with hematologic malignancy, all but 2 are alive. Ten of the 17 with aplasia are alive as are all with thalassemia or sickle cell disease. None of the four variables tested affected survival.
Conclusion:
Our analysis indicates that the standardized preparative regimen, coupled with a now well-established immunosuppressive regimen, is as effective in patients under 18 years of age as in adults. Our analysis also indicates that in a resource-scarce or developing country, it is mandatory to limit high-risk and relatively expensive procedures to active teams that enjoy international accreditation, whether these be in the state or private sector.
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