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  • Just got back from the Ebmt 2019 Adwp report one more study of pediatric patients

    Kirill Kirgizov1,2, Ekaterina Pristanskova1, Elvira Volkova1, Oksana Blagonravova1, Nataliya Sidorova1, Veronika Konstantinova1, Elena Machneva1, Sergey Piliya1, Olga Filina1, Julia Nikolaeva1, Anastasiya Mezentseva1, Raisa Bembeeva1, Elena Skorobogatova1 1The Russian Children’s Research Hospital, Moscow, Russian Federation, 2Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology, Moscow, Russian Federation Background: HSCT for children with multiple sclerosis (MS) proved effectiveness and safety. It is required to improve the results by analysis of long-term follow-up and late effects. Several challenges identified in multidisciplinary collaboration for successful treatment as well as a problem of switching these patients to the adult healthcare. We aimed to create a model of organization of help for children with severe refractory multiple sclerosis based on multidisciplinary and multicenter approach.
    Methods: Fifteen children with MS received autologous HSCT (aHSCT) from January 2010 to May 2018. Gender: females – 11, males – 4. Mean length of MS prior aHSCT was 22.7±8.4 months and mean age of MS debut was 12.7±2.1 years old. All patients had severe refractory MS treated with corticosteroids, interferons, plasmapheresis and mitoxantron with negative results. These patients presented signs of neuroinflammation. Mean baseline EDSS before the start of mobilization was 4.8±1.4. Procedures included mobilization with the help of cyclophosphamide and filgrastim and conditioning: Cyclophosphamide 200 mg/kg and hATG 160 mg/kg. All patients received at least 2 x 10˄6/kg CD34+ hematopoietic stem cells (mean 4.3 x 10˄6±2.6 x 10 ˄6). We analysed the incidence and nature of late effects in patients with at least one year of follow-up (based on the standard protocol for late effects). Fertility preservation proposed for patients. aHSCT as well as pre- and post-transplant care was done by multidisciplinary team involved both transplant and neurological team. Technology of transfer patients to adult center for post-transplant observation was identified.
    Results: All patients survived. Mean time to engraftment was 11 ±1.6 days. Eleven patients experienced culture negative fever, one patient – cystitis, and one patient had CMV reactivation within 100 days of the transplant. No patient experienced an EDSS increase post-HSCT above baseline, and all patients improved. Mean improvement of EDSS was 2.8±1.2 during the first 60 days after aHSCT (fast recovery). In-time transplanted patients improved better. Improvement confirmed by immunological data (increasing of immune regulation index and T-regs in comparison with the baseline). Median follow-up period was 48 months (8-93 months). Four patients (26.7%) experienced exacerbations (both neurological and MRI) in median of 2 years (1-3 years) after aHSCT. No onsets of secondary autoimmune disease and malignancies was seen. Cardiocascular late effects were seen in 6 patients and endocrine – in 3 patients (all females). All these late effects were successfully treated. Patients after the age of 18 years old were transferred to partnering adult center. This center uses the same protocol for HSCT (in adults) and post-transplant observation. Detailed scheme of transfer developed.
    Conclusions: aHSCT for pediatric patients with severe refractory MS appears to be safe and effective method and in-time HSCT can significantly improve the outcomes. Most of the patients did not experienced exacerbations. Late effects found in patients were successfully treated. We provide a best care for these patients in both childhood and adulthood by transferring them to adult center. Thus, a unique multicenter and multidisciplinary model of care for children with severe refractory MS was found.
    Disclosure: Nothing to declare


  • I’m so glad that this is the focus of a meeting to address it and raise its profile; this is a scary concept. Thanks for all the hard work and not this slip away – lets hope its prevalence does not increase in the future, as we seem to be seeing with other kinds of MS

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