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Вопросы гематологии/онкологии и иммунопатологии в педиатрии. 2023; 22: 104-112

Клинический опыт применения даратумумаба в терапии парциальной красноклеточной аплазии после аллогенной трансплантации гемопоэтических стволовых клеток у детей

Аракелян С. К., Благов С. Л., Коврыгин С. И., Семченкова А. А., Фадеева М. С., Евсеев Д. А., Салимова Т. Ю., Байдильдина Д. Д., Шелихова Л. Н., Масчан М. А., Масчан А. А.

https://doi.org/10.24287/1726-1708-2023-22-2-104-112

Аннотация

   Парциальная красноклеточная аплазия (ПККА) – редкое осложнение при AB0-несовместимых аллогенных трансплантациях гемопоэтических стволовых клеток, проявляющееся в частичном или полном отсутствии эритроидных предшественников у реципиентов при удовлетворительной функции других ростков кроветворения. Считается, что лизис эритрокариоцитов при ПККА обусловлен образованием антител остаточными В-лимфоцитами и / или длительно живущими плазматическими клетками реципиента, способными к пролиферации и активной экспрессии маркера СD38. С появлением моноклонального антитела IgG1 к СD38 стала доступна новая потенциально эффективная таргетная терапевтическая опция в отношении рефрактерной ПККА.

   Целью работы является анализ собственных результатов терапии ПККА даратумумабом.

   Родители пациентов дали согласие на использование информации, в том числе фотографий детей, в научных исследованиях и публикациях.

Список литературы

1. Балашов Д. Н. Особенности проведения трансфузионной терапии у пациентов после трансплантации гемопоэтических стволовых клеток. Обзор литературы / Д. Н. Балашов, П. Е. Трахтман // Онкогематология. – 2013. – (3): 42–7.

2. Henig I., Yehudai-Ofir D., Zohar Y., Zuckerman T. Pure Red cell aplasia following ABO-mismatched allogeneic hematopoietic stem cell transplantation: resolution with daratumumab treatment. Acta Haematologica 2021; 144 (6): 683–87.

3. Asawapanumas T., Chanswangphuwana C., Watanaboonyongcharoen P., Rojnuckarin P., Bunworasate U. Daratumumab as a frontline immunosuppression for pure red cell aplasia after major ABO-mismatched allogeneic hematopoietic stem cell transplantation. Leukemia Res Rep 2022; 17: 100314.

4. Jeyaraman P., Borah P., Rajput P., Dayal N., Pathak S., Naithani R. Daratumumab for pure red cell aplasia post ABO incompatible allogeneic hematopoietic stem cell transplant for aplastic anemia. Blood Cells Mol Dis 2021; 88: 102464.

5. Rautenberg C., Kaivers J., Germing U., Haas R., Ackerstaff S., Hoffmann T., et al. Daratumumab for treatment of pure red cell aplasia after allogeneic stem cell transplantation. Bone Marrow Transplant 2020; 55 (6): 1191–3.

6. Кучер М. А. Роль и оптимизация трансфузиологической тактики при трансплантации гемопоэтических стволовых клеток у онкологических и гематологических больных : Дис. … д-ра мед. наук. – СПб., 2018. – С. 27–8.

7. Marco-Ayala J., Gómez-Seguí I., Sanz G., Solves P. Pure red cell aplasia after major or bidirectional ABO incompatible hematopoietic stem cell transplantation: to treat or not to treat, that is the question. Bone Marrow Transplant 2021; 56 (4): 769–78.

8. Longval T., Galimard J. E., Leprêtre A. C., Suarez F., Amiranoff D., Cazaux M., et al. Treatment for pure red cell aplasia after major ABO-incompatible allogeneic stem cell transplantation: a multicentre study. Br J Haematol 2021; 193 (4): 814–26.

9. Лушова А. А. Субпопуляции В-лимфоцитов: функции и молекулярные маркеры / А. А. Лушова [и др.] // Иммунология. – 2019. – 40 (6): 63–76.

10. Сидорова Е. В. Долгоживущие В-клетки / Е. В. Сидорова // Успехи современной биологии. – 2013. – 133 (4): 333–48.

11. Maschan A. A., Skorobogatova E. V., Balashov D. N., Pashanov E. D., Trakhtman P. E., Schipitzina I. P., et al. Successful treatment of pure red cell aplasia with a single dose of rituximab in a child after major ABO incompatible peripheral blood allogeneic stem cell transplantation for acquired aplastic anemia. Bone Marrow Transplant 2002; 30 (6): 405–7.

12. Gómez R. V., Vázquez G. V., Concepción V. N., García A. G., Saavedra C. A. Successful treatment of pure red cell aplasia with high-dose dexamethasone after ABO-incompatible allogeneic hematopoietic stem cell transplantation. Hematol Oncol Stem Cell Ther 2018; 11 (1): 44–6.

13. Kopińska A., Helbig G., Frankiewicz A., Grygoruk-Wiśniowska I., Kyrcz-Krzemień S. Rituximab is highly effective for pure red cell aplasia and post-transplant lymphoproliferative disorder after unrelated hematopoietic stem cell transplantation. Contemp Oncol (Pozn) 2012; 16 (3): 215–7.

14. Chapuy C. I., Kaufman R. M., Alyea E. P., Connors J. M. Daratumumab for delayed red-cell engraftment after allogeneic transplantation. N Engl J Med 2018; 379 (19): 1846–50.

15. Bathini S., Holtzman N. G., Koka R., Singh Z., Wilding E., Zou Y., et al. Refractory postallogeneic stem cell transplant pure red cell aplasia in remission after treatment with daratumumab. Am J Hematol 2019; 94 (8): 216–9.

16. Salas M. Q., Alahmari A., Lipton J. H. Successful treatment of refractory red cell aplasia after allogeneic hematopoietic cell transplantation with daratumumab. Eur J Haematol 2020; 104 (2): 145–7.

17. Martino R., García-Cadenas I., Esquirol A. Daratumumab may be the most effective treatment for post-engraftment pure red cell aplasia due to persistent anti-donor isohemagglutinins after major AB0-mismatched allogeneic transplantation. Bone Marrow Transplant 2022; 57 (2): 282–5.

18. Gangat N., Bleeker J., Lynch D., Olteanu H., Letendre L., Tefferi A. Daratumumab for treatment-refractory acquired idiopathic pure red cell aplasia. Haematologica 2022; 107 (10): 2523–6.

Pediatric Hematology/Oncology and Immunopathology. 2023; 22: 104-112

Daratumumab in the treatment of pure red cell aplasia after pediatric allogeneic stem cell transplantation

Arakelyan S. K., Blagov S. L., Kovrygin S. I., Semchenkova A. A., Fadeeva M. S., Evseev D. A., Salimova T. Yu., Baidildina D. D., Shelikhova L. N., Maschan M. A., Maschan A. A.

https://doi.org/10.24287/1726-1708-2023-22-2-104-112

Abstract

Pure red cell aplasia (PRCA) is a rare complication of AB0-incompatible allogeneic hematopoietic stem cell transplantation, which manifests as a partial or complete absence of erythroid lineage in recipients with normal function of other hematopoietic lineages. There is a hypothesis, that lysis of erythroid precursors occurs because of antibody formation by population of residual B-lymphocytes and/or long-lived recipient’s plasma cells, which are capable for proliferation and active expression of the CD38 marker. That is why the invention of the IgG1 monoclonal antibody to CD38 presented as a new potentially effective targeted therapeutic option for patients with refractory PPCA. The article summarize clinical data on daratumumab for the therapy of PRCA in pediatric allogeneic hematopoietic stem cell transplantation recipients. The patients' parents gave their consent to the use of their children's data, including photographs, for research purposes and in publications.

References

1. Balashov D. N. Osobennosti provedeniya transfuzionnoi terapii u patsientov posle transplantatsii gemopoeticheskikh stvolovykh kletok. Obzor literatury / D. N. Balashov, P. E. Trakhtman // Onkogematologiya. – 2013. – (3): 42–7.

2. Henig I., Yehudai-Ofir D., Zohar Y., Zuckerman T. Pure Red cell aplasia following ABO-mismatched allogeneic hematopoietic stem cell transplantation: resolution with daratumumab treatment. Acta Haematologica 2021; 144 (6): 683–87.

3. Asawapanumas T., Chanswangphuwana C., Watanaboonyongcharoen P., Rojnuckarin P., Bunworasate U. Daratumumab as a frontline immunosuppression for pure red cell aplasia after major ABO-mismatched allogeneic hematopoietic stem cell transplantation. Leukemia Res Rep 2022; 17: 100314.

4. Jeyaraman P., Borah P., Rajput P., Dayal N., Pathak S., Naithani R. Daratumumab for pure red cell aplasia post ABO incompatible allogeneic hematopoietic stem cell transplant for aplastic anemia. Blood Cells Mol Dis 2021; 88: 102464.

5. Rautenberg C., Kaivers J., Germing U., Haas R., Ackerstaff S., Hoffmann T., et al. Daratumumab for treatment of pure red cell aplasia after allogeneic stem cell transplantation. Bone Marrow Transplant 2020; 55 (6): 1191–3.

6. Kucher M. A. Rol' i optimizatsiya transfuziologicheskoi taktiki pri transplantatsii gemopoeticheskikh stvolovykh kletok u onkologicheskikh i gematologicheskikh bol'nykh : Dis. … d-ra med. nauk. – SPb., 2018. – S. 27–8.

7. Marco-Ayala J., Gómez-Seguí I., Sanz G., Solves P. Pure red cell aplasia after major or bidirectional ABO incompatible hematopoietic stem cell transplantation: to treat or not to treat, that is the question. Bone Marrow Transplant 2021; 56 (4): 769–78.

8. Longval T., Galimard J. E., Leprêtre A. C., Suarez F., Amiranoff D., Cazaux M., et al. Treatment for pure red cell aplasia after major ABO-incompatible allogeneic stem cell transplantation: a multicentre study. Br J Haematol 2021; 193 (4): 814–26.

9. Lushova A. A. Subpopulyatsii V-limfotsitov: funktsii i molekulyarnye markery / A. A. Lushova [i dr.] // Immunologiya. – 2019. – 40 (6): 63–76.

10. Sidorova E. V. Dolgozhivushchie V-kletki / E. V. Sidorova // Uspekhi sovremennoi biologii. – 2013. – 133 (4): 333–48.

11. Maschan A. A., Skorobogatova E. V., Balashov D. N., Pashanov E. D., Trakhtman P. E., Schipitzina I. P., et al. Successful treatment of pure red cell aplasia with a single dose of rituximab in a child after major ABO incompatible peripheral blood allogeneic stem cell transplantation for acquired aplastic anemia. Bone Marrow Transplant 2002; 30 (6): 405–7.

12. Gómez R. V., Vázquez G. V., Concepción V. N., García A. G., Saavedra C. A. Successful treatment of pure red cell aplasia with high-dose dexamethasone after ABO-incompatible allogeneic hematopoietic stem cell transplantation. Hematol Oncol Stem Cell Ther 2018; 11 (1): 44–6.

13. Kopińska A., Helbig G., Frankiewicz A., Grygoruk-Wiśniowska I., Kyrcz-Krzemień S. Rituximab is highly effective for pure red cell aplasia and post-transplant lymphoproliferative disorder after unrelated hematopoietic stem cell transplantation. Contemp Oncol (Pozn) 2012; 16 (3): 215–7.

14. Chapuy C. I., Kaufman R. M., Alyea E. P., Connors J. M. Daratumumab for delayed red-cell engraftment after allogeneic transplantation. N Engl J Med 2018; 379 (19): 1846–50.

15. Bathini S., Holtzman N. G., Koka R., Singh Z., Wilding E., Zou Y., et al. Refractory postallogeneic stem cell transplant pure red cell aplasia in remission after treatment with daratumumab. Am J Hematol 2019; 94 (8): 216–9.

16. Salas M. Q., Alahmari A., Lipton J. H. Successful treatment of refractory red cell aplasia after allogeneic hematopoietic cell transplantation with daratumumab. Eur J Haematol 2020; 104 (2): 145–7.

17. Martino R., García-Cadenas I., Esquirol A. Daratumumab may be the most effective treatment for post-engraftment pure red cell aplasia due to persistent anti-donor isohemagglutinins after major AB0-mismatched allogeneic transplantation. Bone Marrow Transplant 2022; 57 (2): 282–5.

18. Gangat N., Bleeker J., Lynch D., Olteanu H., Letendre L., Tefferi A. Daratumumab for treatment-refractory acquired idiopathic pure red cell aplasia. Haematologica 2022; 107 (10): 2523–6.