Hematopoietic stem cell transplantation for non-malignant hematological disorders
Authors:
Renata Formánková; Jan Starý
Authors place of work:
Klinika dětské hematologie a onkologie 2. LF UK a FN Motol, Praha
Published in the journal:
Vnitř Lék 2018; 64(5): 530-536
Category:
Summary
Allogeneic hematopoietic stem cell transplantation (HSCT) is currently successfully used in the treatment of many non-malignant hematopoietic disorders, including acquired and inherited bone marrow failure. HSCT from a HLA-identical sibling donor (MSD) is the treatment of choice, better results are achieved using bone marrow graft. Conditioning regimens and graft-versus-host disease prophylaxis are different in dependence on the underlying disease, age and clinical condition of the patient, type of donor and stem cell graft. Transplantation from MSD is the first-line treatment for children and young adults with SAA. Patients with hemoglobinopathies and inherited bone marrow failure syndromes with available MSD should be referred for transplantation as soon as possible, before the development of serious complications and iron overload-related tissue damage. Indications for HSCT from matched unrelated donor in the treatment of non-malignant hematopoietic disorders are expanding in dependence on very good results in the last two decades. The aim of successfully HSCT is not only to increase the percentage of surviving patients but also to improve the quality of life with the minimum of early complications and late effects.
Key words:
allogeneic hematopoietic stem cell transplantation – aplastic anemia – hemoglobinopathy – inherited bone marrow failure
Zdroje
- Apelbaum FR, Forman SJ, Negrin RS et al. Thomas´ Hematopoietic Cell Transplantation. 4th ed. Blackwell: 2008. ISBN 978–1405153485.
- Cetkovský P, Mayer J, Starý J et al. Transplantace kostní dřeně a periferních hematologických buněk. Galén: Praha 2016. ISBN 978–80–7492–267–1.
- Miano M, Dufour C. The diagnosis and treatment of aplastic anemia: a review. Int J Hematol 2015; 101(6): 527–535. Dostupné z DOI: <http://dx.doi.org/10.1007/s12185–015–1787-z>.
- Singh P, Sinha A, Kamath A et al. Aplastic Anemia – A Quick Review. Cancer Prev Curr Res 2017; 7(5): 00251. Dostupné z DOI: <http://dx.doi.org/10.15406/jcpcr.2017.07.00251>.
- Socié G. Allogeneic BM transplantation fot the treatment of aplastic anemia: current results and expanding donor possibilities. Hematology Am Soc Hematol Educ Program 2013; 2013: 82–86. Dostupné z DOI: <http://dx.doi.org/10.1182/asheducation-2013.1.82>.
- Bacigalupo A, Socié G, Hamladji RM et al. Current outcome of HLA identical sibling versus unrelated donor transplants in severe aplastic anemia: an EBMT analysis. Haematologica 2015; 100(5): 696–702. Dostupné z DOI: <http://dx.doi.org/10.3324/haematol.2014.115345>.
- Bacigalupo A, Socié E, Lanino E et al. Fludarabine, cyclophosphamide, antithymocyte globulin, with or without low dose total body irradiation, for alternative donor transplants, in acquired severe aplastic anemia: a retrospective study from the EBMT-SAA working party. Haematologica 2010; 95(6): 976–982. Dostupné z DOI: <http://dx.doi.org/10.3324/haematol.2009.018267>.
- Dufour C, Pillon M, Socié G et al. Outcome of aplastic anaemia in children. A study by the severe aplastic anaemia and paediatric disease working parties of the European group blood and bone marrow transplant. Br J Haematol 2015; 169(4): 565–573. Dostupné z DOI: <http://dx.doi.org/10.1111/bjh.13297>.
- Marsh JCW, Kulasekararaj AG. Management of the refractory aplastic anemia patient: what are the options? Blood 2013; 122(22): 3561–3567. Dostupné z DOI: <http://dx.doi.org/10.1182/blood-2013–05–498279>.
- Dufour C, Veys P, Carraro E. Similar outcome of upfront-unrelated and matched sibling stem cell transplantation in idiopathic paediatric aplastic anaemia. A study on behalf of the UK Paediatric BMT Working Party, Paediatric Diseases Working Party and Severe Aplastic Anaemia Working Party of EBMT. Br J Haematol 2015; 171(4): 585–594. Dostupné z DOI: <http://dx.doi.org/10.1111/bjh.13614>.
- Brodsky A. Paroxysmal nocturnal hemoglobinuria. Blood 2014; 124(18): 2804–2811. Dostupné z DOI: <http://dx.doi.org/10.1182/blood-2014–02–522128>.
- de Latour RP, Schrezenmeier H, Bacigalupo A et al. Allogeneic stem cell transplantation in paroxysmal nocturnal hemoglobinuria. Haematologica 2012; 97(11): 1666–1673. Dostupné z DOI: <http://dx.doi.org/10.3324/haematol.2012.062828>.
- Angelucci E, Mathes-Martin S, Baronciani D et al. Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an internal expert panel. Haematologica 2014; 99(5): 811–820. Dostupné z DOI: <http://dx.doi.org/10.3324/haematol.2013.099747>.
- Lucarelli G, Isgró A, Sodani P. Hematopoietic Stem Cell transplantation in Thalassemia and Sickle Cell Anemia. Cold Spring Harb Perspect Med 2012; 2(5): a011825. Dostupné z DOI: <http://dx.doi.org/10.1101/cshperspect.a011825>.
- de Latour RP, Peters C, Gibson B et al. Recommendations on hematopoietic stem cell transplantation for inherited bone marrow failure syndromes. Bone Marrow Transplant 2015; 50(9): 1168–1172. Dostupné z DOI: <http://dx.doi.org/10.1038/bmt.2015.117>.
- Dalle JH, de Latour RP Allogeneic hematopoietic stem cell transplantation for inherited bone marrow failure syndromes. Int J Hematol 2016; 103(4): 373–379. Dostupné z DOI: <http://dx.doi.org/10.1007/s12185–016–1951–0>.
- de Latour RP, Porcher R, Dalle JH et al. Allogeneic hematopoietic stem cell transplantation in Fanconi anemia: the European Group for Blood and Marrow Transplantation experience. Blood 2013; 122(26): 4279–4286. Dostupné z DOI: <http://dx.doi.org/10.1182/blood-2013–01–479733>.
- Zecca M, Strocchio L, Pagliara D et al. HLA-Haploidentical T Cell-Depleted Allogeneic Hematopoietic Stem Cell Transplantation in Children with Fanconi Anemia. Biol Blood Marrow Transplant 2014; 20(4): 571–576. Dostupné z DOI: <http://dx.doi.org/10.1016/j.bbmt.2014.01.015>.
- Fioredda F, Iacobelli S, van Biezen A et al. Stem cell transplantation in severe congenital neutropenia: an analysis from the European Society for Blood and Marrow Transplantation. Blood 2015; 126(6): 1885–1892; quiz 1970. Dostupné z DOI: <http://dx.doi.org/10.1182/blood-2015–02–628859>.
- Donadieu J, Fenneteau O, Beaupain B et al. Classification of and risk factors for hematologic complications in a french national cohort of 102 patients with Shwachman-Diamond syndrome. Haematologica 2012; 97(9): 1312–1319. Dostupné z DOI: <http://dx.doi.org/10.3324/haematol.2011.057489>.
- Fagioli F, Quarello P, Zecca M et al. Haematopoietic stem cell transplantation for Diamond Blackfan anemia: a report from the Italian Association of pediatric haematology and oncology registry. Br J Haematol 2014; 165(5): 673–681. Dostupné z DOI: <http://dx.doi.org/10.1111/bjh.12787>.
- Dalle JH, Fahd M. Allegeneic stem cell transplantation in amegacaryocytosis: results of a retrospective study in EBMT centers. Biol Blood Marrow Transplant 2014; 20(2 Suppl): S81-S82. Dostupné z DOI: <https://doi.org/10.1016/j.bbmt.2013.12.099>.
- Gadalla SM, Sales-Bonfim C, Carreras J et al. Outcomes of allogeneic hematopoietic cell transplantation in patients with dyskeratosis congenita. Biol Blood Marrow Transplant 2013; 19(8): 1238–1243. Dostupné z DOI: <http://dx.doi.org/10.1016/j.bbmt.2013.05.021>.
- Unal S, Russo R, Gumruk F et al. Successful hematopoietic stem cell transplantation in a patient with congenital dyserythropoietic anemia type II. Pediatr Transplant 2014; 18(4): E130-E133. Dostupné z DOI: <http://dx.doi.org/10.1111/petr.12254>.
Štítky
Diabetology Endocrinology Internal medicineČlánok vyšiel v časopise
Internal Medicine
2018 Číslo 5
Najčítanejšie v tomto čísle
- Autoimmune hemolytic anemia
- Differential diagnosis of anemia
- Congenital and acquired bleeding disorders
- Hemoglobinopathies