Вопросы гематологии/онкологии и иммунопатологии в педиатрии. 2024; 23: 14-22
Эпидемиология приобретенной апластической анемии у детей в Российской Федерации
https://doi.org/10.24287/1726-1708-2024-23-4-14-22Аннотация
Приобретенная апластическая анемия (ПАА) во всем мире остается редким заболеванием. Однако частота встречаемости ее в разных странах крайне вариабельна. В настоящей статье представлены результаты эпидемиологического исследования ПАА у детей в Российской Федерации. Исследование одобрено независимым этическим комитетом и утверждено решением ученого совета ФГБУ «НМИЦ ДГОИ им. Дмитрия Рогачева» Минздрава России. Выполнен анализ эпидемиологических данных у пациентов с ПАА в возрасте от 0 до 18 лет, проходивших дистанционную диагностику в лабораториях ФГБУ «НМИЦ ДГОИ им. Дмитрия Рогачева» Минздрава России в период с 1 сентября 2017 г. по 31 декабря 2023 г., в рамках впервые проведенного в нашей стране регистрового исследования. В анализ вошли 582 пациента из 77 регионов Российской Федерации, охват детской популяции составил 97%. Средний показатель заболеваемости за 6 лет составил 3,1 случая на 1 млн детского населения в год. Медиана возраста пациентов – 10,2 года (диапазон 1–17,9 года). Большинство случаев ПАА приходилось на возрастные периоды 5–9 и 10–14 лет. В возрасте до 1 года не выявлено ни одного случая заболевания. Мальчиков было больше, чем девочек: 317 (54,5%) и 265 (45,5%) соответственно. По этиологии у 87% пациентов диагностирована идиопатическая ПАА, у 13% – гепатит-ассоциированная ПАА. Критерии сверхтяжелой формы ПАА выполнялись у 45,7% пациентов, тяжелой – у 38,8% и нетяжелой – у 15,5%. Сочетание ПАА с клоном пароксизмальной ночной гемоглобинурии диагностировано у 42,3% педиатрических пациентов. По показателю заболеваемости ПАА у детей Российская Федерация занимает срединное место между странами Европы и Азии. Основные проанализированные демографические показатели и характеристики заболевания сопоставимы с данными мировой литературы.
Список литературы
1. Hartung Н.D., Olson T.S., Bessler M. Acquired aplastic anemia in children. Pediatr Clin North Am 2013; 60 (6): 1311–36.
2. Montane E., Ibanez L., Vidal X., Ballarín E., Puig R., García N., et al. Epidemiology of aplastic anemia: a prospective multicenter study. Haematologica 2008; 93: 518–23.
3. Young N.S. Aplastic Anemia. N Engl J Med 2018; 379 (17): 1643–56.
4. Young N.S., Scheinberg P., Calado R.T. Aplastic Anemia. Curr Opin Hematol 2008; 15 (3): 162–8.
5. Gonzalez-Casas R., Garcia-Buey L., Jones E.A., Gisbert J.P., Moreno-Otero R. Systematic review: hepatitis-associated aplastic anaemia – a syndrome associated with abnormal immunological function. Aliment Pharmacol Ther 2009; 30 (5): 436–43.
6. Camitta B.M., Rappeport J.M., Parkman R., Nathan D.G. Selection of patients for bone marrow transplantation in severe aplastic anemia. Blood 1975; 45: 355–63.
7. Jeong Dae Chul, Chung Nack Gyun, Kang Hyoung Jin, Koo Hong Hoe, Kook Hoon, Kim Soon Ki, et al. Epidemiology and clinical long-term outcome of childhood aplastic anemia in Korea for 15 years: retrospective study of the Korean Society of Pediatric Hematology Oncology (KSPHO). Pediatr Hematol Oncol 2011; 33 (3): 172–8.
8. Young N.S., Kaufman D.W. The epidemiology of acquired aplastic anemia. Haematologica 2008; 93 (4): 489–92.
9. Кугейко Т.Б., Быданов О.И. Приобретенная апластическая анемия у детей в Республике Беларусь: эпидемиология и этиология. Вопросы гематологии/онкологии и иммунопатологии в педиатрии 2018; 17 (1): 29–36. DOI: 10.24287/1726-1708-2018-17-1-29-36 [
10. Kulagin A., Lisukov I., Ivanova M., Golubovskaya I., Kruchkova I., Bondarenko S., et al. Prognostic value of paroxysmal nocturnal haemoglobinuria clone presence in aplastic anaemia patients treated with combined immunosuppression: results of two-centre prospective study. Br J Haematol 2014; 164 (4): 546–54.
11. Горонкова О.В., Тузова Е.А., Салимова Т.Ю., Фоминых В.С., Шутова А.Д., Мехиева Э.Х. и соавт. Дифференциальная диагностика приобретенной апластической анемии у детей: анализ данных регистрового исследования. Педиатрия. Журнал им. Г.Н. Сперанского 2024; 103 (3): 8–20. DOI: 10.24110/0031-403X-2024-103-3-8-20
12. Горонкова О.В., Павлова А.В., Райкина Е.В. Апластическая анемия у детей: современная концепция дифференциальной диагностики. Вопросы гематологии/онкологии и иммунопатологии в педиатрии 2024; 23 (2): 208–20. DOI: 10.24287/1726-1708-2024-23-2-208-220
13. Sipol A.A., Babenko E.V., Borisov V.I., Naumova E.V., Boyakova E.V., Yakunin D.I., et al. An inter-laboratory comparison of PNH clone detection by high-sensitivity flow cytometry in a Russian cohort. Hematology 2015; 20 (1): 31–8.
14. Kojima S. Why is the incidence of aplastic anemia higher in Asia? Expert Rev Hematol 2017; 10 (4): 277–9.
15. Issaragrisil S., Kaufman D.W., Anderson T., Chansung K., Leaverton P.E., Shapiro S., et al. The epidemiology of aplastic anemia in Thailand. Blood 2006; 107 (4): 1299–307.
16. Ahmed P., Chaudhry Q.U.N., Satti T.M., Mahmood S.K., Ghafoor T., Shahbaz N., et al. Epidemiology of aplastic anemia: a study of 1324 cases. Hematology 2020; 25 (1): 48–54.
17. Wire M.B., Li X., Zhang J., Sallas W., Aslanis V., Ouatas T. Modeling and simulation support eltrombopag dosing in pediatric patients with immune thrombocytopenia. Clin Pharmacol Ther 2018; 104: 1199–207.
18. Rauff B., Idrees M., Shah S.A., Butt S., Butt A.M., Ali L., et al. Hepatitis associated aplastic anemia: a review. Virol J 2011; 8: 87.
19. Osugi Y., Yagasaki H., Sako M., Kosaka Y., Taga T., Ito T., et al. Japan Childhood Aplastic Anemia Study Group. Antithymocyte globulin and cyclosporine for treatment of 44 children with hepatitis associated aplastic anemia. Haematologica 2007; 92 (12): 1687–90.
20. Locasciulli A., Bacigalupo A., Bruno B., Montante B., Marsh J., Tichelli A., et al. Hepatitis associated aplastic anemia: Epidemiology and treatment results obtained in Europe. A report of the EBMT aplastic anaemia working party. Br J Haematol 2010; 149: 890–5.
21. Fu L., Zhang J., Wang R., Yang B., Li H., Chen H., et al. Clinical characteristics of hepatitis-associated aplastic anemia in children. Ann Hematol 2024; 103 (2): 397–404.
22. Altay D., Yýlmaz E., Özcan A., Karakükçü M., Ünal E., Arslan D. Hepatitis-associated aplastic anemia in pediatric patients: single center experience. Transfus Apher Sci 2020; 59: 102900.
23. Bharadwaj R., Menon J., Kumar V., Shanmugam N., Munirathnam D. Clinical Profile of Hepatitis Associated Aplastic Anemia (HAAA) in Six Children. Indian Pediatr 2023; 60 (5): 410–1.
24. Yang W., Zhao X., Peng G., Zhang L., Jing L., Zhou K., et al. Time and residual hematopoiesis are crucial for PNH clones escape in hepatitis-associated aplastic anemia. Ann Hematol 2021; 100 (10): 2435–41.
Pediatric Hematology/Oncology and Immunopathology. 2024; 23: 14-22
Epidemiology of acquired aplastic anemia in children in the Russian Federation
https://doi.org/10.24287/1726-1708-2024-23-4-14-22Abstract
Although acquired aplastic anemia (AA) remains a rare disease worldwide, the incidence of acquired AA in different countries varies widely. This article presents the results of an epidemiological study of acquired AA in children in the Russian Federation. The study was approved by the Independent Ethics Committee and the Scientific Council of the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation. In this study, we analyzed the epidemiological data of patients aged 0 to 18 years with acquired AA who had been diagnosed remotely at the laboratories of the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology from September 1, 2017 to December 31, 2023 as part of the first registry study conducted in Russian Federation. Our analysis included data from 582 patients from 77 regions of the Russian Federation; the coverage of the pediatric population was 97%. The average incidence rate for the 6-year period was 3.1 cases per 1 million children per year. The median age was 10.2 years (range 1–17.9 years). Most cases of acquired AA occurred between the ages of 5 and 9 years and between 10 and 14 years. There were no cases of acquired AA in children under the age of 1 year. There were more males than females: 317 (54.5%) versus 265 (45.5%). According to etiology, 87% of the patients were diagnosed with idiopathic AA, and 13% had hepatitis-associated AA. In total, 45.7% of the patients fulfilled the criteria for very severe AA, 38.8% for severe AA, and 15.5% for non-severe AA. Paroxysmal nocturnal hemoglobinuria clone was detected in 42.3% of the patients with acquired AA. The incidence rate of acquired AA in children in Russian Federation is intermediate between the European and Asian incidence rates. Our main demographic data and disease characteristics are consistent with the world data.
References
1. Hartung N.D., Olson T.S., Bessler M. Acquired aplastic anemia in children. Pediatr Clin North Am 2013; 60 (6): 1311–36.
2. Montane E., Ibanez L., Vidal X., Ballarín E., Puig R., García N., et al. Epidemiology of aplastic anemia: a prospective multicenter study. Haematologica 2008; 93: 518–23.
3. Young N.S. Aplastic Anemia. N Engl J Med 2018; 379 (17): 1643–56.
4. Young N.S., Scheinberg P., Calado R.T. Aplastic Anemia. Curr Opin Hematol 2008; 15 (3): 162–8.
5. Gonzalez-Casas R., Garcia-Buey L., Jones E.A., Gisbert J.P., Moreno-Otero R. Systematic review: hepatitis-associated aplastic anaemia – a syndrome associated with abnormal immunological function. Aliment Pharmacol Ther 2009; 30 (5): 436–43.
6. Camitta B.M., Rappeport J.M., Parkman R., Nathan D.G. Selection of patients for bone marrow transplantation in severe aplastic anemia. Blood 1975; 45: 355–63.
7. Jeong Dae Chul, Chung Nack Gyun, Kang Hyoung Jin, Koo Hong Hoe, Kook Hoon, Kim Soon Ki, et al. Epidemiology and clinical long-term outcome of childhood aplastic anemia in Korea for 15 years: retrospective study of the Korean Society of Pediatric Hematology Oncology (KSPHO). Pediatr Hematol Oncol 2011; 33 (3): 172–8.
8. Young N.S., Kaufman D.W. The epidemiology of acquired aplastic anemia. Haematologica 2008; 93 (4): 489–92.
9. Kugeiko T.B., Bydanov O.I. Priobretennaya aplasticheskaya anemiya u detei v Respublike Belarus': epidemiologiya i etiologiya. Voprosy gematologii/onkologii i immunopatologii v pediatrii 2018; 17 (1): 29–36. DOI: 10.24287/1726-1708-2018-17-1-29-36 [
10. Kulagin A., Lisukov I., Ivanova M., Golubovskaya I., Kruchkova I., Bondarenko S., et al. Prognostic value of paroxysmal nocturnal haemoglobinuria clone presence in aplastic anaemia patients treated with combined immunosuppression: results of two-centre prospective study. Br J Haematol 2014; 164 (4): 546–54.
11. Goronkova O.V., Tuzova E.A., Salimova T.Yu., Fominykh V.S., Shutova A.D., Mekhieva E.Kh. i soavt. Differentsial'naya diagnostika priobretennoi aplasticheskoi anemii u detei: analiz dannykh registrovogo issledovaniya. Pediatriya. Zhurnal im. G.N. Speranskogo 2024; 103 (3): 8–20. DOI: 10.24110/0031-403X-2024-103-3-8-20
12. Goronkova O.V., Pavlova A.V., Raikina E.V. Aplasticheskaya anemiya u detei: sovremennaya kontseptsiya differentsial'noi diagnostiki. Voprosy gematologii/onkologii i immunopatologii v pediatrii 2024; 23 (2): 208–20. DOI: 10.24287/1726-1708-2024-23-2-208-220
13. Sipol A.A., Babenko E.V., Borisov V.I., Naumova E.V., Boyakova E.V., Yakunin D.I., et al. An inter-laboratory comparison of PNH clone detection by high-sensitivity flow cytometry in a Russian cohort. Hematology 2015; 20 (1): 31–8.
14. Kojima S. Why is the incidence of aplastic anemia higher in Asia? Expert Rev Hematol 2017; 10 (4): 277–9.
15. Issaragrisil S., Kaufman D.W., Anderson T., Chansung K., Leaverton P.E., Shapiro S., et al. The epidemiology of aplastic anemia in Thailand. Blood 2006; 107 (4): 1299–307.
16. Ahmed P., Chaudhry Q.U.N., Satti T.M., Mahmood S.K., Ghafoor T., Shahbaz N., et al. Epidemiology of aplastic anemia: a study of 1324 cases. Hematology 2020; 25 (1): 48–54.
17. Wire M.B., Li X., Zhang J., Sallas W., Aslanis V., Ouatas T. Modeling and simulation support eltrombopag dosing in pediatric patients with immune thrombocytopenia. Clin Pharmacol Ther 2018; 104: 1199–207.
18. Rauff B., Idrees M., Shah S.A., Butt S., Butt A.M., Ali L., et al. Hepatitis associated aplastic anemia: a review. Virol J 2011; 8: 87.
19. Osugi Y., Yagasaki H., Sako M., Kosaka Y., Taga T., Ito T., et al. Japan Childhood Aplastic Anemia Study Group. Antithymocyte globulin and cyclosporine for treatment of 44 children with hepatitis associated aplastic anemia. Haematologica 2007; 92 (12): 1687–90.
20. Locasciulli A., Bacigalupo A., Bruno B., Montante B., Marsh J., Tichelli A., et al. Hepatitis associated aplastic anemia: Epidemiology and treatment results obtained in Europe. A report of the EBMT aplastic anaemia working party. Br J Haematol 2010; 149: 890–5.
21. Fu L., Zhang J., Wang R., Yang B., Li H., Chen H., et al. Clinical characteristics of hepatitis-associated aplastic anemia in children. Ann Hematol 2024; 103 (2): 397–404.
22. Altay D., Yýlmaz E., Özcan A., Karakükçü M., Ünal E., Arslan D. Hepatitis-associated aplastic anemia in pediatric patients: single center experience. Transfus Apher Sci 2020; 59: 102900.
23. Bharadwaj R., Menon J., Kumar V., Shanmugam N., Munirathnam D. Clinical Profile of Hepatitis Associated Aplastic Anemia (HAAA) in Six Children. Indian Pediatr 2023; 60 (5): 410–1.
24. Yang W., Zhao X., Peng G., Zhang L., Jing L., Zhou K., et al. Time and residual hematopoiesis are crucial for PNH clones escape in hepatitis-associated aplastic anemia. Ann Hematol 2021; 100 (10): 2435–41.
