Email this article Sirolimus efficacy in treatment of autoimmune lymphoproliferative syndrome
- Authors: Shvetz O.A.1, Deripapa E.V.1, Abramova I.N.1, Victorova E.A.1, Rodina Y.A.1, Deordieva E.A.1, Baidildina D.D.1, Kalinina I.I.1, Petrova U.N.1, Pshonkin A.V.1, Kuzmenko N.B.1, Maschan A.A.1, Shcherbina A.Y.1
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Affiliations:
- Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology
- Issue: Vol 17, No 1 (2018)
- Pages: 46-53
- Section: Статьи
- Submitted: 09.08.2018
- Published: 09.02.2018
- URL: https://hemoncim.com/jour/article/view/26
- DOI: https://doi.org/10.24287/1726-1708-2018-17-1-46-53
- ID: 26
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Abstract
Autoimmune lymphoproliferative syndrome is a primary immunodeficiency caused by defects in proteins participating in the Fas-mediated apoptosis pathway, manifesting with benign and malignant lymphoproliferation and immune cytopenias. Sirolimus is an inhibitor of mammalian target of rapamycin (mTOR-I), it regulates cell growth and proliferation. In this article, we describe the efficacy and safety of sirolimus treatment in a group of 22 patients with ALPS. We demonstrate the effectiveness of therapy, both in the usual dose of 2–3 mg/m2, and in reduced dose of 1–2 mg/m2. Treatment with sirolimus not only leads to the resolution of the symptoms of lymphoproliferation and cytopenias, but also to the normalization of biomarkers, for example, the vitamin B12 level and double negative T-lymphocytes.
About the authors
O. A. Shvetz
Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology
Email: shv18081979@gmail.com
ORCID iD: 0000-0001-5347-7150
MD, pediatrician allergologist/immunologist, outpatient department
Russia 117997, Moscow, Samory Mashela st., 1
+7 (495) 287-6570, ext. 1172
E. V. Deripapa
Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology
Email: shv18081979@gmail.com
ORCID iD: 0000-0002-9083-4783
Russian Federation
I. N. Abramova
Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology
Email: shv18081979@gmail.com
Russian Federation
E. A. Victorova
Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology
Email: shv18081979@gmail.com
ORCID iD: 0000-0002-2427-1417
Russian Federation
Yu. A. Rodina
Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology
Email: shv18081979@gmail.com
Russian Federation
E. A. Deordieva
Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology
Email: shv18081979@gmail.com
ORCID iD: 0000-0002-8208-2075
Russian Federation
D. D. Baidildina
Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology
Email: shv18081979@gmail.com
Russian Federation
I. I. Kalinina
Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology
Email: shv18081979@gmail.com
ORCID iD: 0000-0002-0813-5626
Russian Federation
U. N. Petrova
Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology
Email: shv18081979@gmail.com
ORCID iD: 0000-0002-1258-8281
Russian Federation
A. V. Pshonkin
Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology
Email: shv18081979@gmail.com
ORCID iD: 0000-0002-2057-2036
Russian Federation
N. B. Kuzmenko
Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology
Email: shv18081979@gmail.com
ORCID iD: 0000-0002-1669-8621
Russian Federation
A. A. Maschan
Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology
Email: shv18081979@gmail.com
ORCID iD: 0000-0002-0016-6698
Russian Federation
A. Yu. Shcherbina
Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology
Author for correspondence.
Email: shv18081979@gmail.com
ORCID iD: 0000-0002-3113-4939
Russian Federation
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