García GA, Hernández CC, Carnot UJ, Cepero LK, Pardo RIK, Allué FL

Language: Spanish
References: 20
Page:
PDF size: 350.89 Kb.

ABSTRACT

Introduction: Acquired severe marrow aplasia is a rare hematological disease characterized by decrease or absence of hematopoietic precursors in bone marrow, which is expressed with different degrees of cytopenias. Several factors, infectious or not, can influence its origin. Its management is complex and may include immunosuppressive treatment and allogeneic hematopoietic stem-cell transplantation.
Objective: To demonstrate the usefulness of performing haploidentical allogeneic hematopoietic stem-cell transplantation in patients with severe medullary aplasia.
Clinical case: A 21-year-old male patient, with medical history, who first presented, in October 2018, with icterus, pancytopenia, as well as purpuric hemorrhagic lesions on the skin and mucosa, in the course of acute seronegative hepatitis. The bone marrow biopsy showed severe marrow aplasia. Immunosuppressive treatment was started with antithymocytic globulin, cyclosporine A, and methylprednisolone. After six months, he maintained severe thrombocytopenia under transfusion requirements and, in October 2019, the decision was to perform allogeneic hematopoietic stem-cell transplantation with a haploidentical donor and using antithymocyte globulin, fludarabine, cyclophosphamide, and low doses of total body irradiation as conditioning treatment. In the clinical assessment carried out in July 2020 (day +280 after transplantation), the patient was asymptomatic and with normal hematological parameters.
Conclusions: Transplantation of haploidentic allogeneic hematopoietic progenitors was shown to be a feasible and useful procedure in patients with severe marrow aplasia, which corroborates the clinical benefit that its execution can bring in patients with this disease.

REFERENCES

1. Brodsky RA, Jones RJ. Aplastic anaemia. Lancet. 2005;365:1647-56.

2. Scheinberg P, Young NS. How I treat acquired aplastic anemia. Blood. 2012;120:1185-96.

3. Iamonico E, Camps F, Populin M, Vega A, Meccico M, Gutiérrez M, et al. Hepatitis asociada a aplasia medular. Rev Arg Med. 2017;5(1):55-66.

4. Young N. Current concepts in the pathophysiology and treatment of aplastic anemia. Hematology Am Soc Hematol Educ Program. 2013;2013(1):76-81.

5. Bacigalupo A. How I treat acquired aplastic anemia. Blood. 2017;129 (11):1428-36.

6. Gragert L, Eapen M, Williams E, Freeman J, Spellman S, Baitty R, et al. HLA match likelihoods for hematopoietic stem-cell grafts in the U.S. registry. N Engl J Med. 2014;371:339-48.

7. Dehn J, Arora M, Spellman S, Setterholm M, Horowitz M, Confer D, et al. Unrelated donor hematopoietic cell transplantation: Factors associated with a better HLA match. Biol Blood Marrow Transplant. 2008;14:1334-40.

8. Hernández Cruz C, Carnot Uria J, Muñío Perurena J, Cepero Llauger K, Pardo Ramírez IK. El trasplante alogénico haploidéntico: un traje hecho a la medida de nuestras condiciones y necesidades. Rev Cub Med. 2017;56(1):69-74.

9. Iturrioz Mata A, Guerrero Pereda R, Palacio Pina M, Ruiz de la Hermosa Sosa J, Albisu Andrade Y. Hepatitis y anemia aplásica [Hepatitis and aplastic anemia]. An Pediatr. 2003;58(4):393-4.

10. Rauff B, Idrees M, Shah SA, Butt S, Butt AM, Ali L, et al. Hepatitis associated aplastic anemia: a review. Virol J. 2011;8:87.

11. Liu L, Wang X, Jin S, Hao L, Zhang Y, Zhang X, et al. Haploidentical hematopoietic stem cell transplantation for non-responders to immunosuppressive therapy against acquired severe aplastic anemia. Bone Marrow Transplant. 2016;51(3):424-7.

12. DeZern AE, Zahurak M, Symons H, Cooke K, Jones RJ, Brodsky RA. Alternative Donor Transplantation with High-Dose Post-Transplantation Cyclophosphamide for Refractory Severe Aplastic Anemia. Biol Blood Marrow Transplant. 2017;23(3):498-504.

13. Kim H, Lee JH, Joo YD, Bae SH, Lee SM, Jo JC, et al. Comparable Allogeneic Hematopoietic Cell Transplantation Outcome of a Haplo-Identical Family Donor with an Alternative Donor in Adult Aplastic Anemia. Acta Haematol. 2016;136(3):129-39.

14. Zhang Y, Wu L, Mo W, Zhou M, Li Y, Chen X, et al. Comparable Outcomes of First-Line Hematopoietic Stem Cell Transplantation from Unrelated and Matched Sibling Donors in Adult Patients with Aplastic Anemia: A Retrospective Single-Center Study. Biol Blood Marrow Transplant. 2019;25(8):1567-75.

15. Xu LP, Jin S, Wang SQ, Xia LH, Bai H, Gao SJ, et al. Upfront haploidentical transplant for acquired severe aplastic anemia: registrybased comparison with matched related transplant. J Hematol Oncol. 2017;10(1):25.

16. Clay J, Kulasekararaj AG, Potter V, Grimaldi F, McLornan D, Raj K, et al. Nonmyeloablative peripheral blood haploidentical stem cell transplantation for refractory severe aplastic anemia. Biol Blood Marrow Transplant. 2014;20(11):1711-6.

17. DeZern AE, Zahurak M, Cooke KR, Margolis D, Jones RJ, Brodsky RA; The Path to Cure: Using Haploidentical (haplo) Donors and High-Dose Post-Transplant Cyclophosphamide (PTCy) for Treatment-Naïve and Refractory Severe Aplastic Anemia (SAA). Blood. 2019;134(Supplement_1): 147. doi: 10.1182/blood-2019-121900

18. Bacigalupo A, Giammarco S. Haploidentical donor transplants for severe aplastic anemia. Semin Hematol. 2019;56(3):190-3.

19. Hernández Cruz C, Carnot Uria J, Diego de la Campa J, Muñío Perurena J, Cepero Llauger K, Pardo Ramírez IK, et al. Trasplante de progenitores hematopoyéticos en el Hospital "Hermanos Ameijeiras", 2014-2018. Rev Cub Hematol Inmunol Hemoter. 2018 [citado: 14 Abr 2020 ];34(4):[aprox. 0 p.]. Disponible en: Disponible en: http://www.revhematologia.sld.cu/index.php/hih/article/view/975

20. Hernández Cruz C, Diego de la Campa J, Perurena Muñ?o J, Cepero Llauger K, Pardo Ramirez IK, García Garc?a A, et al. Implementation of Haploidentical Stem Cell Transplantation for Adult Patients in Cuba. Biol Blood Marrow Transplant. 2019; 25(3):323.