Objectives Allogeneic haematopoietic stem cell transplantation (allo‐HSCT) is the only currently available curative treatment for sickle cell disease (SCD). Here, we comprehensively evaluated the reconstitution of T‐ and B‐cell compartments in 29 SCD patients treated with allo‐HSCT and how it correlated with the development of acute graft‐versus‐host disease (aGvHD). Methods T‐cell neogenesis was assessed by quantification of signal‐joint and β‐chain TCR excision circles. B‐cell neogenesis was evaluated by quantification of signal‐joint and coding‐joint K‐chain recombination excision circles. T‐ and B‐cell peripheral subset numbers were assessed by flow cytometry. Results Before allo‐HSCT (baseline), T‐cell neogenesis was normal in SCD patients compared with age‐, gender‐ and ethnicity‐matched healthy controls. Following allo‐HSCT, T‐cell neogenesis declined but was fully restored to healthy control levels at one year post‐transplantation. Peripheral T‐cell subset counts were fully restored only at 24 months post‐transplantation. Occurrence of acute graft‐versus‐host disease (aGvHD) transiently affected T‐ and B‐cell neogenesis and overall reconstitution of T‐ and B‐cell peripheral subsets. B‐cell neogenesis was significantly higher in SCD patients at baseline than in healthy controls, remaining high throughout the follow‐up after allo‐HSCT. Notably, after transplantation SCD patients showed increased frequencies of IL‐10‐producing B‐regulatory cells and IgM + memory B‐cell subsets compared with baseline levels and with healthy controls. Conclusion Our findings revealed that the T‐ and B‐cell compartments were normally reconstituted in SCD patients after allo‐HSCT. In addition, the increase of IL‐10‐producing B‐regulatory cells may contribute to improve immune regulation and homeostasis after transplantation.
BackgroundSystemic sclerosis (SSc) is characterised by skin thickening and visceral involvement, leading to impairment of physical function, daily life activities and quality of life. Severe cases usually have poor prognosis, despite conventional immunosuppressive treatment. Autologous hematopoietic stem cell transplantation (AHSCT) has been investigated as treatment for patients with severe SSc and promotes reduction of skin thickening and at least stabilisation of visceral involvement. There are no reports in the literature addressing the influence of AHSCT on hand function and physical capacity of SSc patients.ObjectivesTo evaluate the impact of AHSCT on skin involvement, hand function, physical capacity and quality of life (QoL) of SSc patients.MethodsThis is a prospective longitudinal study of a cohort of 27 SSc patients who underwent AHSCT at a University Hospital in the state of São Paulo, Brazil. Patients were evaluated before, and at 6, 12 and 24 months after transplant. The evaluations included modified Rodnan skin score (mRSS), hand function (hand-grip strength, finger-to-palm distance – FTP, range of motion measures, DASH and COCHIN questionnaires), mouth opening, six-minute walk test (6MWT) and quality of life questionnaire (SF-36). Results were subjected to statistical analyses and significance levels were established at p<0.05.ResultsTwenty-seven patients were evaluated before and at 6 months after transplant, 22 of which were additionally evaluated at 12 months, and 13 patients at 24 months post-transplant. At 12 months after AHSCT, patients presented significant improvement of mRSS (p<0.01), hand grip strength (p<0.01), range of motion of hands (p<0.01, except for I metacarpophalangeal joints of both hands), FTP distance (p<0.01), DASH (p<0.01), COCHIN (p<0.01), mouth opening (p<0.01), 6MWT distance (p=0.01), and physical (p<0.01) and mental (p=0.02) components of the SF-36, when compared to pre-transplant evaluations. Significant correlations were observed between skin involvement and range of motion measures (dominant hand: R=−0.65, p<0.01; non-dominant hand: R=−0.59, p<0.01), and between the 6MWT distance and quality of life (R=0.62, p<0.01), and between DASH and quality of life (R=−0.48, p=0.03).ConclusionsAHSCT enhances the functional status of SSc patients, significantly improving skin involvement, hand function, physical capacity and quality of life. These results can be interpreted as positive outcomes of AHSCT for SSc.References[1] van Laar, et al. JAMA2014;311(24):2490–8.[2] Del Papa, et al. Bone Marrow Transplantation2017;52:53–58.[3] Burt, et al. Lancet2013:381(9872):1116–24.[4] Sullivan, et al. N Engl J Med2018;378:35–47.Disclosure of InterestNone declared
BackgroundAutologous Hematopoietic Stem Cell Transplantation (AHSCT) has been explored as a therapeutic option for patients with systemic sclerosis (SSc) that do not respond to conventional treatment.ObjectivesTo investigate changes in quality of life of severe and rapidly progressive SSc patients treated with AHSCT.MethodsThis is a longitudinal and comparative study. Patients were evaluated before (n=27), and at 6 (n=27) and 12 months (n=21) after AHSCT. The Generic Questionnaire for Evaluation of Quality of Life Medical Outcomes Study 36 Item Short-Form Health Survey (SF-36) was applied individually, face-to-face, under patient written consent. This questionnaire evaluates eight domains: physical functioning (PF), role-physical (RP), bodily pain (BP), general health (GH), social functioning (SF), vitality (VT), role-emotional (RE) and mental health (MH). Results were transformed into a 0–100 scale, where zero corresponds to a worse health condition and 100 to the best possible score, and submitted to statistical analysis. Significance was defined as p<0.05.ResultsMost participants were females (n=24), with mean age of 33 years (standard deviation, SD=10.33) and mean time from diagnosis of 34.4 months (SD=34.89). Before AHSCT, the mostly impaired aspects were: PF (mean=8.33, SD=18.34), and RP (mean=38.52, SD=21.56), while MH (mean=61.63, SD=15.46) and SF (mean=56.87, SD=27.17) were mostly preserved. At 6 and 12 months post-AHSCT, there was significant improvement of the SF-36 scores in the following domains: PF (6 months, p<0.01, 12 months, p<0.01); RP (6 months, p<0.01, 12 months, p<0.01); BP (6 months, p<0.01, 12 months, p<0.01); GH (6 months, p<0.01, 12 months, p=0.02); VT (6 months, p<0.01, 12 months, p<0.01); MH (6 months, p<0.01, 12 months, p<0.01). The SF domain showed significant increase only at 12 months (p=0.02). The only domain in which there was no significant change was RE.ConclusionsIncreases in the physical components of quality of life are more evident in the initial periods that follow AHSCT, while improvements in mental state, which are also associated with social aspects, are detected on longer follow-up. These data reinforce the relevance of AHSCT upon patient quality of life, signalling the importance of psychotherapeutic evaluations and follow-up.Disclosure of InterestNone declared
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