Fludarabine-Based Reduced-Intensity Conditioning Regimen for Hematopoietic Stem Cell Transplantation in a Pediatric Patient with Sickle Cell Disease: A Case Report
Abstract
Reduced-intensity conditioning (RIC) regimens have the potential to decrease toxicities related to hematopoietic stem cell transplantation (HCT) in patients with sickle cell disease (SCD). While initial results may have been acceptable in adults and young adults, there are no well-established strategies in children with SCD. Here, it is described the clinical course of two children with symptomatic SCD who have successfully undergone HSCT using Fludarabin-based conditioning.
1.Kassim AA, Sharma D. Hematopoietic stem cell transplantation for sickle cell disease: The changing landscape. Hematol Oncol Stem Cell Ther. 2017;10(4):259-266.
2. Guilcher GMT, Truong TH, Saraf SL, et al. Curative therapies: Allogeneic hematopoietic cell transplantation from matched related donors using myeloablative, reduced intensity, and nonmyeloablative conditioning in sickle cell disease. Semin Hematol. 2018;55(2):87-93.
3. Vassal G, Michel G, Espérou H, et al. Prospective validation of a novel IV busulfan fixed dosing for paediatric patients to improve therapeutic AUC targeting without drug monitoring. Cancer Chemother Pharmacol. 2008;61(1):113-23.
4. Matthes-Martin S, Lawitschka A, Fritsch G, et al. Stem cell transplantation after reduced-intensity conditioning for sickle cell disease. Eur J Haematol. 2013;90(4):308-12.
5. Bhatia M, Jin Z, Baker C, et al. Reduced toxicity, myeloablative conditioning with BU, fludarabine, alemtuzumab and SCT from sibling donors in children with sickle cell disease. Bone Marrow Transplant. 2014;49(7):913-20.
6. King AA, Kamani N, Bunin N, et al. Successful matched sibling donor marrow transplantation following reduced intensity conditioning in children with hemoglobinopathies. Am J Hematol. 2015;90(12):1093-8
7. Guilcher GMT, Monagel DA, Nettel-Aguirre A, et al. Nonmyeloablative Matched Sibling Donor Hematopoietic Cell Transplantation in Children and Adolescents with Sickle Cell Disease. Biol Blood Marrow Transplant. 2019;25(6):1179-1186.
8. Ngwube A, Shah N, Godder K, et al. Abatacept is effective as GVHD prophylaxis in unrelated donor stem cell transplantation for children with severe sickle cell disease. Blood Adv. 2020;4(16):3894-3899.
9. Hsieh MM, Fitzhugh CD, Weitzel RP, et al. Nonmyeloablative HLA-matched sibling allogeneic hematopoietic stem cell transplantation for severe sickle cell phenotype. JAMA. 2014;312(1):48-56.
10. Madden LM, Hayashi RJ, Chan KW, et al. Long-Term Follow-Up after Reduced-Intensity Conditioning and Stem Cell Transplantation for Childhood Nonmalignant Disorders. Biol Blood Marrow Transplant. 2016;22(8):1467-1472.
11. Schetelig J, Bornhäuser M, Kiehl M, et al. Reduced-intensity conditioning with busulfan and fludarabine with or without antithymocyte globulin in HLA-identical sibling transplantation--a retrospective analysis. Bone Marrow Transplant. 2004;33(5):483-90.
12. Horn B, Baxter-Lowe LA, Englert L, et al. Reduced intensity conditioning using intravenous busulfan, fludarabine and rabbit ATG for children with nonmalignant disorders and CML. Bone Marrow Transplant. 2006;37(3):263-9.
13. Lee KH, Lee JH, Lee JH, et al. Reduced-Intensity Conditioning with Busulfan, Fludarabine, and Antithymocyte Globulin for Hematopoietic Cell Transplantation from Unrelated or Haploidentical Family Donors in Patients with Acute Myeloid Leukemia in Remission. Biol Blood Marrow Transplant. 2017;23(9):1555-1566.
14. Jacobsohn DA, Duerst R, Tse W, et al. Reduced intensity haemopoietic stem-cell transplantation for treatment of non-malignant diseases in children. Lancet. 2004;364(9429):156-62.
15. Güngör T, Teira P, Slatter M, et al. Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study. Lancet. 2014;383(9915):436-48.
16. Felber M, Steward CG, Kentouche K, et al. Targeted busulfan-based reduced-intensity conditioning and HLA-matched HSCT cure hemophagocytic lymphohistiocytosis. Blood Adv. 2020;4(9):1998-2010.
2. Guilcher GMT, Truong TH, Saraf SL, et al. Curative therapies: Allogeneic hematopoietic cell transplantation from matched related donors using myeloablative, reduced intensity, and nonmyeloablative conditioning in sickle cell disease. Semin Hematol. 2018;55(2):87-93.
3. Vassal G, Michel G, Espérou H, et al. Prospective validation of a novel IV busulfan fixed dosing for paediatric patients to improve therapeutic AUC targeting without drug monitoring. Cancer Chemother Pharmacol. 2008;61(1):113-23.
4. Matthes-Martin S, Lawitschka A, Fritsch G, et al. Stem cell transplantation after reduced-intensity conditioning for sickle cell disease. Eur J Haematol. 2013;90(4):308-12.
5. Bhatia M, Jin Z, Baker C, et al. Reduced toxicity, myeloablative conditioning with BU, fludarabine, alemtuzumab and SCT from sibling donors in children with sickle cell disease. Bone Marrow Transplant. 2014;49(7):913-20.
6. King AA, Kamani N, Bunin N, et al. Successful matched sibling donor marrow transplantation following reduced intensity conditioning in children with hemoglobinopathies. Am J Hematol. 2015;90(12):1093-8
7. Guilcher GMT, Monagel DA, Nettel-Aguirre A, et al. Nonmyeloablative Matched Sibling Donor Hematopoietic Cell Transplantation in Children and Adolescents with Sickle Cell Disease. Biol Blood Marrow Transplant. 2019;25(6):1179-1186.
8. Ngwube A, Shah N, Godder K, et al. Abatacept is effective as GVHD prophylaxis in unrelated donor stem cell transplantation for children with severe sickle cell disease. Blood Adv. 2020;4(16):3894-3899.
9. Hsieh MM, Fitzhugh CD, Weitzel RP, et al. Nonmyeloablative HLA-matched sibling allogeneic hematopoietic stem cell transplantation for severe sickle cell phenotype. JAMA. 2014;312(1):48-56.
10. Madden LM, Hayashi RJ, Chan KW, et al. Long-Term Follow-Up after Reduced-Intensity Conditioning and Stem Cell Transplantation for Childhood Nonmalignant Disorders. Biol Blood Marrow Transplant. 2016;22(8):1467-1472.
11. Schetelig J, Bornhäuser M, Kiehl M, et al. Reduced-intensity conditioning with busulfan and fludarabine with or without antithymocyte globulin in HLA-identical sibling transplantation--a retrospective analysis. Bone Marrow Transplant. 2004;33(5):483-90.
12. Horn B, Baxter-Lowe LA, Englert L, et al. Reduced intensity conditioning using intravenous busulfan, fludarabine and rabbit ATG for children with nonmalignant disorders and CML. Bone Marrow Transplant. 2006;37(3):263-9.
13. Lee KH, Lee JH, Lee JH, et al. Reduced-Intensity Conditioning with Busulfan, Fludarabine, and Antithymocyte Globulin for Hematopoietic Cell Transplantation from Unrelated or Haploidentical Family Donors in Patients with Acute Myeloid Leukemia in Remission. Biol Blood Marrow Transplant. 2017;23(9):1555-1566.
14. Jacobsohn DA, Duerst R, Tse W, et al. Reduced intensity haemopoietic stem-cell transplantation for treatment of non-malignant diseases in children. Lancet. 2004;364(9429):156-62.
15. Güngör T, Teira P, Slatter M, et al. Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study. Lancet. 2014;383(9915):436-48.
16. Felber M, Steward CG, Kentouche K, et al. Targeted busulfan-based reduced-intensity conditioning and HLA-matched HSCT cure hemophagocytic lymphohistiocytosis. Blood Adv. 2020;4(9):1998-2010.
| Files | ||
| Issue | Vol 18, No 1 (2024) | |
| Section | Case Report(s) | |
| DOI | https://doi.org/10.18502/ijhoscr.v18i1.14750 | |
| Keywords | ||
| Reduced-intensity conditioning; Children; Nonmalignant disorders; Sickle cell disease | ||
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This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License. |
How to Cite
1.
Builes N. Fludarabine-Based Reduced-Intensity Conditioning Regimen for Hematopoietic Stem Cell Transplantation in a Pediatric Patient with Sickle Cell Disease: A Case Report. Int J Hematol Oncol Stem Cell Res. 2024;18(1):104-109.
