Autoimmune diseases of children: what can be learned from the experience of pediatric hematologist and oncologists?

Introduction. Today, the problem of therapy of autoimmune diseases has acquired high relevance. The strategy of achievement of success is interaction of doctors of various specializations. One of the advanced approaches to treatment of this group of diseases is joining of efforts with hematologists and oncologists.

A variant of such interaction is an example of successful treatment of children with refractory forms of multiple sclerosis or cell therapy with the use of autologous transplantation of hematopoietic stem cells. The article represents a brief review of therapy of autoimmune diseases with the focus on observation of patients with multiple sclerosis.

Materials and methods. 13 patients with multiple sclerosis were included into the analysis. Distribution of sexes: 9 girls (69.2 %) and 4 boys (30.8 %). Age median: 16.7 ± 1.7 years. The median of duration of the disease prior to treatment is 15.5 ± 4.1 years. The age of onset of the disease is 4 to 14 years (12.3 ± 1.7 years). The average point in accordance with the Expanded Disability Status Scale (EDSS) is 6.16 ± 0.2 points. A severe refractory course of disease was registered with all patients. Until the moment of commencement of the therapy with the methods represented in the article, the patients received treatment with corticosteroids, interferon, plasmapheresis, and mitoxantrone with negative results. All patients in the group of autologous transplantation commenced the therapy with the signs of autoimmune inflammation. Transfusions of ex vivo cultivated T-regulatory cells were applied in the context of protocol regarding the cell therapy. The conditioning scheme with the use of the combination of cyclophosphamide (200 mg / kg) and atgam drug (160 mg / kg) was used in the case of performance of autologous transplantation.

Results. Remission without aggravation episodes was maintained with the patients that received therapy with autologous T-regulatory cells. Rapid improvement in the early post-transplantation period was registered with all children after auto-TGSC. The maximum improvement under the EDSS scale comprised 5.5 points. The article represented the experience of long-term observation of patients of this group (the maximum observation period is 60 months). No serious side effects were registered as a result of the therapy. Only 2 patients at late terms demonstrated repeated aggravation of the disease.

Conclusions. Thus, the presented methods of therapy demonstrated their efficiency in the context of refractory forms of multiple sclerosis and can be translated to other types of autoimmune diseases.

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