Immunosupressive therapy of aplastic anemia patients: successes and failures (single center experiment 2007–2016)

E. A. Mikhaylova; Михайлова Елена Алексеевна; Z. T. Fidarova; Фидарова Залина Таймуразовна; A. V. Abramova; Абрамова Анастасия Владимировна; A. V. Luchkin; Лучкин Антон Владимирович; V. V. Troitskaya; Троицкая Вера Витальевна; V. N. Dvirnyk; Двирных Валентина Николаевна; I. V. Galtseva; Гальцева Ирина Владимировна; G. A. Kliasova; Клясова Галина Александровна; A. M. Kovrigina; Ковригина Алла Михайловна; S. M. Kulikov; Куликов Сергей Михайлович; Yu. А. Chabaeva; Чабаева Юлия Александровна; E. N. Parovichnikova; Паровичникова Елена Николаевна; V. G. Savchenko; Савченко Валерий Григорьевич; T. N. Obukhova; Обухова Татьяна Никифоровна

doi:10.26442/00403660.2020.07.000756

Immunosupressive therapy of aplastic anemia patients: successes and failures (single center experiment 2007–2016)

Authors: Mikhaylova E.A.¹, Fidarova Z.T.¹, Abramova A.V.¹, Luchkin A.V.¹, Troitskaya V.V.¹, Dvirnyk V.N.¹, Galtseva I.V.¹, Kliasova G.A.¹, Kovrigina A.M.¹, Kulikov S.M.¹, Chabaeva Y.А.¹, Parovichnikova E.N.¹, Savchenko V.G.¹, Obukhova T.N.¹
Affiliations:
1. National Research Center for Hematology
Issue: Vol 92, No 7 (2020)
Pages: 4-9
Section: Editorial
URL: https://journals.rcsi.science/0040-3660/article/view/43117
DOI: https://doi.org/10.26442/00403660.2020.07.000756
ID: 43117

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Abstract

Treatment programs for patients with acquired aplastic anemia include two main therapeutic options: allogeneic bone marrow transplantation and combined immunosuppressive therapy (IST). However, combined IST remains the method of choice for most adult AA patients. This study included 120 AA patients who received IST at the National Research Center for Hematology in 2007–2016. The analysis was applied to 120 patients. Median age was 25 (17–65) years, M/F: 66/54, SAA/NSAA: 66%/34%. Effectiveness of IST was carried out in 120 patients with AA. This group did not include 8 SAA patients who died during the first 3 months from the start of treatment from severe infectious complications (early deaths – 6.2%) and 2 AA patients who dropped out of surveillance. The observation time was 55 (6–120) months. Paroxysmal nocturnal hemoglobinuria (PNH clone) was detected in 67% of AA patients. The median PNH clone size (granulocytes) was 2.5 (0.01–99.5)%. The treatment was according to the classical protocol of combined IST: horse antithymocytic globulin and cyclosporin A. Most of patients (87%) responded to combined immunosuppressive therapy. To achieve a positive response, it was sufficient to conduct one course of ATG to 64% of patients, two courses of ATG – 24% of patients and 2% of patients responded only after the third course of ATG. A positive response after the first course was obtained in 64% of patients included in the analysis. Most of the responding patients (93%) achieve a positive response after 3–6 months from the start of treatment. Therefore, the 3rd–6th months after the first course of ATG in the absence of an answer to the first line of therapy can be considered the optimal time for the second course of ATG. This tactic allows to get an answer in another 58% of patients who did not respond to the first course of ATG. The probability of an overall 10-year survival rate was 90% (95% confidence interval 83.6–96.2).

Keywords

idiopathic aplastic anemia, acquired aplastic anemia, immunosuppressive therapy, antithymocyte globulin, paroxysmal nocturnal hemoglobinuria, cyclosporin A