Haematopoietic stem cell transplantation in paediatric patients with myeloid malignancies; 15 years of experience
Keywords:
Haematopoietic stem cell transplantation, Myeloid leukaemia, Myelodysplastic syndrome, Survival, InfectionAbstract
Objective: To describe the experience, in terms of survival and complications, with patients that received a haematopoietic stem cell transplantation (HSCT) in the last 15 years.
Materials and methods: A descriptive, retrospective, cohort study was conducted on patients less than 18 years-old with a diagnosis of acute myeloid leukaemia (AML), chronic myeloid leukaemia (CML), and myelodysplastic syndrome (MDS) and received HSCT between January 2001 and December 2015. The outcomes were, overall survival, event-free survival, and complications associated with the transplant.
Results: During the study period, a total of 43 patients with myeloid neoplasms received a transplant, of which 31 (72%) had AML, 4 (9%) with CML, and 8 (19%) with MDS. A total of 11 identical donor transplants were performed, as well as 11 haploidentical, 11 autologous, and 10 with umbilical cord blood. Of the patients with AML (31 cases), 58% were subjected to a transplant in the first full remission, and 39% in the second or subsequent full remission. The overall and event-free survival at 5 years was 56% and 38%, respectively. The transplant-related mortality at day 100 was 15%, with an accumulated incidence of graft versus host disease of 59%, cytomegalovirus infection of 39%, with 54% bacterial infections, and 14% haemorrhagic cystitis.
Conclusion: The low mortality and complications rate associated with the transplant suggests that haematopoietic stem cell transplantation is a viable alternative as a treatment for patients with myeloid neoplasms in our country.
Author Biographies
Diego Medina, Fundación Valle del Lili
Hemato-oncología pediátrica y trasplante de médula ósea, Fundación Valle del Lili, Cali, Colombia
Lina Gonzales, Universidad Icesi
Facultad de Ciencias de la Salud, Departamento de salud pública, Universidad Icesi, Cali, Colombia
Diana Davalos, Universidad Icesi
Facultad de Ciencias de la Salud, Departamento de salud pública, Universidad Icesi, Cali, Colombia
María Rosales , Fundación Valle del Lili
Trasplante de médula ósea, Fundación Valle del Lili, Cali, Colombia
María Rosales , Fundación Valle del Lili
Trasplante de médula ósea, Fundación Valle del Lili, Cali, Colombia
Eliana Manzi, Fundación Valle del Lili
Centro de investigaciones clínicas, Fundación Valle del Lili, Cali, Colombia
Mayra Estacio, Fundación Valle del Lili
Centro de investigaciones clínicas, Fundación Valle del Lili, Cali, Colombia
References
Redner A, Kessel R, Acute Myeloid Leukemia. In: Lanzkowsky P, Lipton JM, Fish JD. Lanzkowsky's Manual of Pediatric Hematology and Oncology. 6 th Edition. Elsevier. 390-406.
https://doi.org/10.1016/B978-0-12-801368-7.00019-3
Puumala SE, Ross JA, Aplenc R, Spector LG. Epidemiology of childhood acute myeloid leukemia. Pediatr Blood Cancer. 2013;60:728-33.
https://doi.org/10.1002/pbc.24464
Pinkel D. ''Allogeneic marrow transplantation in children with acute leukemia: a practice whose time has gone'': twenty years later. Leukemia. 2009;23:2189-96.
https://doi.org/10.1038/leu.2009.132
Flower A, Cairo MS. The evolution of allogeneic stem cell transplant for children and adolescents with acute myeloid leukemia. Clin Adv Hematol Oncol. 2017;15:52-62.
Rau ATK, Shreedhara AK, Kumar S. Myelodysplastic syndromes in children: where are we today? Ochsner J. 2012;12:216-20.
Rubnitz JE. Current Management of Childhood Acute Myeloid Leukemia. Pediatr Drugs. 2017;19:1-10.
https://doi.org/10.1007/s40272-016-0200-6
Hijiya N, Schultz KR, Metzler M, Millot F, Suttorp M. Pediatric chronic myeloid leukemia is a unique disease that requires a different approach. Blood. 2016;127:392-9.
https://doi.org/10.1182/blood-2015-06-648667
Gluckman E. Choice of the donor according to HLA typing and stem cell source. En: Apperley J, Carreras E, Gluckman E, Masszi T, editores. EBMT-ESH Handbook on Haematopoietic Stem Cell Transplantation. 6 th ed. Geneva, Switzerland: EBMT-ESH (European Group for Blood and Marrow Transplantation - European School of Haematology; 2012. p. 176-95.
Bacigalupo A, Ballen K, Rizzo D, Giralt S, Lazarus H, Ho V, Apperley J, et al. Defining the intensity of conditioning regimens: working definitions. Biol Blood Marrow Transplant. 2009;15:1628-33.
https://doi.org/10.1016/j.bbmt.2009.07.004
Sanz J, Picardi A, Hernández Boluda JC, Martín C, Ferrá C, Nozzoli C, et al. Impact of graft-versus-host disease prophylaxis on outcomes after myeloablative single-Unit umbilical cord blood transplantation. Biol Blood Marrow Transplant. 2013;19:1387-92.
https://doi.org/10.1016/j.bbmt.2013.07.004
Jacobsohn DA, Vogelsang GB. Acute graft versus host disease. Orphanet J Rare Dis. 2007;2:35.
https://doi.org/10.1186/1750-1172-2-35
Mrsi'c M, Labar B, Bogdani'c V, Nemet D, Pavleti'c Z, Plavsi'c F, et al. Combination of cyclosporin and methotrexate for prophylaxis of acute graft-versus-host disease after allogeneic bone marrow transplantation for leukemia. Bone Marrow Transplant. 1990;6:137-41.
Ringdén O, Klaesson S, Sundberg B, Ljungman P, Lönnqvist B, Persson U. Decreased incidence of graft-versushost disease and improved survival with methotrexate combined with cyclosporin compared with monotherapy in recipients of bone marrow from donors other than HLA identical siblings. Bone Marrow Transplant. 1992;9:19-25.
Klein OR, Buddenbaum J, Tucker N, Chen AR, Gamper CJ, Loeb D, et al. Nonmyeloablative Haploidentical Bone Marrow Transplantation with Post-Transplantation Cyclophosphamide for Pediatric and Young Adult Patients with High-Risk Hematologic Malignancies. Biol Blood Marrow Transplant. 2017;23: 325-32.
https://doi.org/10.1016/j.bbmt.2016.11.016
Robinson TM, O'Donnell PV, Fuchs EJ, Luznik L. Haploidentical bone marrow and stem cell transplantation: experience with post-transplantation cyclophosphamide. Semin Hematol. 2016;53:90-7.
https://doi.org/10.1053/j.seminhematol.2016.01.005
Luznik L, O'Donnell PV, Fuchs EJ. Post-transplantation cyclophosphamide for tolerance induction in HLA-haploidentical bone marrow transplantation. Semin Oncol. 2012;39: 683-93.
https://doi.org/10.1053/j.seminoncol.2012.09.005
Tomblyn M, Chiller T, Einsele H, Gress R, Sepkowitz K, Storek J, et al. Guidelines for preventing infectious complications among hematopoietic cell transplantation recipients: a global perspective. Biol Blood Marrow Transplant. 2009;15: 1143 -238.
https://doi.org/10.1016/j.bbmt.2009.06.019
Kim HT, Armand P. Clinical Endpoints in allogeneic hematopoietic stem cell transplantation studies: the cost of freedom. Biol Blood Marrow Transplant. 2013;19:860-6.
https://doi.org/10.1016/j.bbmt.2013.01.003
Przepiorka D, Weisdorf D, Martin P, Klingemann HG, Beatty P, Hows J, et al. 1994 Consensus Conference on Acute GVHD Grading. Bone Marrow Transplant. 1995;15:825-8.
Szydlo R. Statistical evaluation of HSCT data. In: EBMT-ESH Handbook on Haematopoietic Stem Cell Transplantation. 6 thed. Geneva, Switzerland: EBMT-ESH (European Group for Blood and Marrow Transplantation - European School of Haematology); 2012. p. 613-28.
Nesbit ME, Buckley JD, Feig SA, Anderson JR, Lampkin B, Bernstein ID, et al. Chemotherapy for induction of remission of childhood acute myeloid leukemia followed by marrow transplantation or multiagent chemotherapy: a report from the Childrens Cancer Group. J Clin Oncol. 1994;12: 127-35.
https://doi.org/10.1200/JCO.1994.12.1.127
Dahl GV, Kalwinsky DK, Mirro J, Look AT, Pui CH, Murphy SB, et al. Allogeneic bone marrow transplantation in a program of intensive sequential chemotherapy for children and young adults with acute nonlymphocytic leukemia in first remission. J Clin Oncol. 1990;8:295-303.
https://doi.org/10.1200/JCO.1990.8.2.295
Marjerrison S, Antillon F, Bonilla M, Fu L, Martinez R, Valverde P, et al. Outcome of children treated for relapsed acute myeloid leukemia in Central America. Pediatr Blood Cancer. 2014;61:1222-6.
https://doi.org/10.1002/pbc.24942
Woods WG, Neudorf S, Gold S, Sanders J, Buckley JD, Barnard DR, et al. A comparison of allogeneic bone marrow transplantation, autologous bone marrow transplantation, and aggressive chemotherapy in children with acute myeloid leukemia in remission. Blood. 2001;97:56-62.
https://doi.org/10.1182/blood.V97.1.56
Wayne AS, Baird K, Egeler RM. Hematopoietic stem cell transplantation for leukemia. Pediatr Clin North Am. 2010;57: 1-25.
https://doi.org/10.1016/j.pcl.2009.11.005
Jaiswal SR, Chakrabarti S. Haploidentical transplantation in children with acute leukemia: The unresolved issues. Adv Hematol. 2016;2016:3467672.
https://doi.org/10.1155/2016/3467672
Fabricius WA, Ramanathan M. Review on haploidentical hematopoietic cell transplantation in patients with hematologic malignancies. Adv Hematol. 2016;2016:5726132.
https://doi.org/10.1155/2016/5726132
Mancusi A, Ruggeri L, Velardi A. Haploidentical hematopoietic transplantation for the cure of leukemia: from its biology to clinical translation. Blood. 2016;128:2616-23.
https://doi.org/10.1182/blood-2016-07-730564
Nassereddine S, Rafei H, Elbahesh E, Tabbara I. Acute graft versus host disease: A comprehensive review. Anticancer Res. 2017;37:1547-55.
https://doi.org/10.21873/anticanres.11483
Jacobsohn DA, Vogelsang GB. Acute graft versus host disease. Orphanet J Rare Dis. 2007;2:35.
https://doi.org/10.1186/1750-1172-2-35
Zittoun RA, Mandelli F, Willemze R, de Witte T, Labar B, Resegotti L, et al. Autologous or allogeneic bone marrow transplantation compared with intensive chemotherapy in acute myelogenous leukemia. N Engl J Med. 1995;332:217-23.
How to Cite
Downloads
Downloads
Published
Issue
Section
License
Todos los derechos reservados.
