Successful Outcomes of Pediatric Hematopoietic Cell Transplantations for Thalassemia and Sickle Cell Diseases

Main Article Content

Preeda Vanichsetakul, MD

Abstract

OBJECTIVE: To assess outcomes of hematopoietic cell transplantation (HCT) for thalassemias and hemoglobinopathies in a single medical center in Thailand.


METHODS: Case series study for thalassemia and hemoglobinopathy patients undergoing HCT at Bangkok Hospital Medical Center (BMC) from February 2009 through December 2013.


RESULTS: There were a total of 15 patients. Eleven cases were Thais, 1 was French-Thai, 1 was Bangladeshi, 1 was Lao, and 1 patient was Omani who had sickle cell disease (SCD). Thirteen cases were diagnosed as beta-thalassemia/hemoglobin E diseases, 1 as a transfusion-dependent alpha-thalassemia, and 1 as SCD. Among the 15 HCTs, 9 patients underwent bone marrow transplant (BMT), 4 patients underwent umbilical cord blood transplant (CBT), and 2 patients underwent combined cord blood and marrow transplantation (CB+BMT). All donors were related and fully-HLA-matched. The male to female patient ratio was 12:3. The patients’ ages at transplant varied from 2 years to 15 years 11 months with a median of 5 years. The patient’s body weight varied from 11.1 kilogram (kg) to 50kg (a median of 17.3 kg). According to the Psaro classification, among the 14 thalassemia patients there were 10 class I, and 4 class II patients. Busulfan, fludaradine, and rabbit ATG were mainly used as the myeloablative conditioning regimen. Cyclosporine and short-course methotrexate were mainly used as graft-versus-host disease (GvHD) Prophylaxis in BMT and CB+BMT groups, while cyclosporine alone was used in the CBT group. CD34+ cell doses per kilogram body weight recipients ranged from 5.6 to 34.7x10^6 (a median of 12.3x10^6) in the BMT group (n=9), and from 1.6 to 3x10^5 (median 2.3x10^5) in the CBT group (n=4). Complete donor engraftments were achieved in 12 patients. Mixed-chimerism states with donor predominance were present in 2 patients from the BMT group and 1 patient from the CBT group. No patients experienced graft failure. Neutrophil recoveries were evident on days +10 to +23 (median day +14), and platelet recoveries were observed on days +19 to +64 (median day +40). No patients developed acute or chronic GvHD. There were no mortalities. Median follow up tine for all patients was 2 years 2 months (1 month to 4 years 10 months). Overall (OS) and disease-free survival (DFS) were 100% and 100% for all patients (n=15). Based on the risk class, the OS and DFS for class I thalassemia patients (n=10) were 100% and 100%, and class II patients (n=4) were 100% and 100%, respectively


CONCLUSION: Our experience in HCT for thalassemias and hemoglobinopathies has been very favorable. HLA-matched related donor HCT yielded the best success rate. Anyhow, regular follow-up visits are encouraged to detect any possible complications and to determine long term outcomes.

Downloads

Download data is not yet available.

Article Details

How to Cite
1.
Vanichsetakul P. Successful Outcomes of Pediatric Hematopoietic Cell Transplantations for Thalassemia and Sickle Cell Diseases. BKK Med J [Internet]. 2014Feb.20 [cited 2020Aug.6];7(1):1. Available from: https://he02.tci-thaijo.org/index.php/bkkmedj/article/view/218717
Section
Original Article

References

1. Lucarelli G, Isgro A, Sodani P, et al. Hematopoietic stem cell transplantation in thalassemia and sickle cell anemia. Cold Spring Harb Perspect Med 2012;2:a011825.
2. Angelucci E. Hematopoietic Stem Cell Transplantation in Thalassemia. ASH Education Book 2010;1:456-62.
3. Thomas ED, Buckner CD, Sanders JE, et al. Marrow transplantation for thalassaemia. Lancet 1982;2:227-9.
4. VanichsetakulP,WacharaprechanontT,O-CharoenR,etal. Umbilical cord blood transplantation in children with beta-thalassemia diseases. J Med Assoc Thai 2004;87:S62-7.
5. Old JM, Olivieri NF, Thein SL. Diagnosisandmanagement of thalassaemia. In: Weatherall DJ, Clegg JB eds. The Thalassaemia Syndromes, 4th ed. Oxford: Blackwell Science Ltd., 2001:663.
6. Rocha V, Wagner JE, Sobocinski KA, et al. Graft-versus- host disease in children who have received a cord blood or bone marrow transplant from an HLA-identical sibling. Eurocord and international bone marrow trans- plant registry working committee on alternative donor and stem cell sources. N Engl J Med 2000;342:1846-54.
7. Gluckman E. Hematopoietic stem cell transplants using umbilical cord blood. N Engl J Med 2001;344:1860-1.
8. WacharaprechanontT,O-CharoenR,VanichsetakulP,etal. Cord blood collection for the National Cord Blood Bank in Thailand. J Med Assoc Thai 2003;86:S409-16.
9. Ryu SG, Lee JH, Choi SJ,et al. Randomized comparison of four-times-dailyversusonce dailyintravenousbusulfan in conditioning therapy for hematopoietic cell transplan- tation. Biol Blood Marrow Transplant 2007;13:1095-105.
10. Nguyen L, Fuller D, Lennon S, et al. I.V. busulfan in pediatrics: a novel dosing to improve safety/efficacy for hematopoietic progenitor cell transplantation recipients. Bone Marrow Transplant 2004;33:979-87.
11. Vogelsang GB, Hess AD. Graft-versus-host disease: new directions for a persistent problem. Blood 1994;84:2061-7.
12. Glucksberg H, Storb R, Fefer A, et al. Clinical mani- festations of graft-versus-host disease in human recipients of marrow from HLA-matched sibling donors. Transplan- tation 1974;18:295-304.
13. Storb R, Prentice RL, Sullivan KM, et al. Predictive factors in chronic graft-versus-host disease in patients with aplastic anemia treated by bone marrow transplan- tation from HLA-identical siblings. Ann Intern Med 1983; 98:461-6.
14. Locatelli F, Percivalle E, Comoli P, et al. Human cyto megalovirus (HCMV) infection in pediatric patients- given allogeneic bone marrow transplantation: role of early treatment of antigenemia on patients’ outcome. Br J Haematol 1994;88:64-71.
15. Lucarelli G, Weatherall DJ. For debate: bone marrow transplantation for severe thalassaemia (1). The view from Pesaro (2). To be or not to be. Br J Haematol 1991;78:300-3.
16. Lucarelli G, Polchi P, Izzi T, et al. Allogeneic marrow transplantation for thalassemia. Exp Hematol 1984; 12:676-81.
17. Gaziev J, Sodani P, Lucarelli G. Hematopoietic stem cell transplantation in thalassemia. Bone Marrow Transplan- tation 2008;42:S41.