Objectives To assess the value of rituximab (RTM) in a limited series of patients (pt) with juvenile systemic sclerosis (jSSc).
Methods Three patients with jSSc were evaluated: 2 girls (ptA and ptB), 1 boy (ptC) with onset at 7, 8 and 7 years, and duration of the disease at the beginning of therapy 4, 6 and 7 years, all with diffuse form of jSSc. Patients had a high activity and aggressive course of the disease with predominant pulmonary (interstitial lung disease – ptA, ptB), heart (ptC), microcirculatory disturbances (ptA, ptB), typical and severe skin lesions and musculoskeletal system, immunological activity (ANA “+” - all, aScl70 “+” - ptA and ptB, ACA “+” – ptC). Therapy, previous RTM, included corticosteroids, cyclophosphamide, blockers ACE alprostadil. They received RTM at a dose of 375 mg/m2 every 6 months; ptA received 4 infusions, pnB – 3, ptC – 6, with a total duration of use of RTM 24, 18 and 36 months. The use of RTM approved by local ethics committee.
Results Improvement was noted in all patients. The first manifestations of improvement established after 6 months of the RTM treatment with subsequent increase. All patients showed marked improvement in health, increased exercise tolerance, reduction of skin tightening (after 6 months of therapy there was a decrease of the Rodnan total skin thickness score, 6 points; with a further decrease in accordance with the duration of the treatment), sclerodactyly, improving joint function (polyarthropathies), a gradual increase in DLCO, FVC (6-8%), indicators of heart disease (ptC, with a dominant extremely serious cardiac disease, reducing the size of the left ventricular end-diastolic dimension, from 58 to 52 mm, increase in ejection fraction from 32 to 48%, reducing the number of ventricular ectopic beats per day from 19,657 to 1,671). During the period of treatment revealed no significant adverse events associated with the use of rituximab.
Conclusions The results of our observations of small number of patients with severe jSSc who had RTM therapy suggests that the use of RTM is a promising treatment for jSS with the presence of a good therapeutic effect, with an acceptable adverse event profile, reduces the need for glucocorticoids, limit the use of cytotoxic drugs. Further studies of the efficacy of this therapy in the treatment of jSSc are needed.
Disclosure of Interest None declared
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