Background/Objective We sought to evaluate if autologous hematopoietic stem cell transplantation (AHSCT) influences the functional status of systemic sclerosis (SSc) patients. Methods From 2014 to 2018, a cohort of 27 SSc patients was assessed before, and at 6 and 12 months after AHSCT for modified Rodnan’s skin score (mRSS), mouth opening, hand grip strength, range of motion (ROM), functional ability of upper limbs (DASH questionnaire and Cochin hand function scale—CHFS), 6-minute walk test (6MWT), and quality of life (SF-36 questionnaire). Linear regression models with random effects and Spearman’s test were used for statistical analysis. Results At 6 and 12 months after AHSCT, respectively, we observed significant improvement of mRSS (p < 0.01 and p < 0.01), mouth opening (p = 0.02 and p < 0.01), hand function (DASH, p < 0.01 and p < 0.01; CHFS, p < 0.01 and p < 0.01; strength, p < 0.01 and p < 0.01), physical capacity (6MWT, p = 0.02 and p = 0.03) and physical (p < 0.01 and p < 0.01) and mental (ns and p = 0.02) component scores of SF-36. At 12 months after AHSCT, ROM measurements improved (p < 0.05) in five out of six evaluated joints in both hands, compared to baseline. Correlation was significant between physical capacity and quality of life (R = 0.62; p < 0.01), between DASH and quality of life (R = −0.48; p = 0.03), and between skin involvement and wrist ROM measures (dominant hand, R = −0.65, p < 0.01; non-dominant hand, R = −0.59; p < 0.01). Conclusions AHSCT enhances the functional status of SSc patients in the first year of follow-up, significantly improving hand function, physical capacity and quality of life. These results are interpreted as positive outcomes of AHSCT for SSc.
Objective: describe the development of a virtual learning object to provide information about autologous transplantation of hematopoietic stem cells to autoimmune diseases. Methods: methodological study of a website development, using the instructional design model that includes Analysis, Design, Development and Implementation. Results: the virtual object, available at http://www.transplantardai.com.br, was developed in a web platform, in the Hypertext Markup Language, using the software WebAcappella -Responsive Website Creator (Intuisphere, France 2016). The content was structured in the modules: History, Transplant, Autoimmune Diseases, Links, Guidelines, Speech Team and Doubts. The icons and menus were created in order to attract the user, facilitating the search for information and allowing maximum use of the resources available on the website. Conclusion: the methodology used allowed the development of the virtual learning object, which can be used as a tool to guide and disseminate knowledge about this treatment. RESUMOObjetivo: descrever o desenvolvimento de um objeto virtual de aprendizagem para disponibilização de informações sobre transplante autólogo de células-tronco hematopoéticas para doenças autoimunes. Métodos: estudo metodológico de desenvolvimento de um website, empregando o modelo de design instrucional que envolve Análise, Design, Desenvolvimento e Implementação. Resultados: o objeto virtual, disponível no endereço eletrônico http://www.transplantardai.com.br, foi desenvolvido em plataforma web, na linguagem de marcação Hypertext Markup Language, utilizando-se o software WebAcappella -Responsive Website Creator (Intuisphere, França 2016). O conteúdo foi estruturado nos seguintes módulos: História, Transplante, Doenças Autoimunes, Links, Orientações, Fala Equipe e Dúvidas. Os ícones e menus foram criados de modo a atrair o usuário, facilitando a busca de informações e permitindo máximo uso dos recursos disponíveis no website. Conclusão: a metodologia empregada permitiu o desenvolvimento do objeto virtual de aprendizagem, que poderá ser utilizado como ferramenta para orientar e disseminar o conhecimento sobre esse tratamento. Descritores: Transplante de Células-Tronco Hematopoéticas; Doenças Autoimunes; Materiais de Ensino; Educação a Distância; Internet. RESUMENObjetivo: describir el desarrollo de un objeto de aprendizaje virtual para proporcionar información sobre el trasplante autólogo de células madre hematopoyéticas en las enfermedades autoinmunes. Métodos: estudio metodológico del desarrollo del sitio web, utilizando el modelo de diseño instruccional (Análisis, Diseño, Desarrollo e Implementación). Resultados: el objeto virtual, disponible en http://www.transplantardai.com.br, fue desarrollado en una plataforma web, en el lenguaje de marcación Hypertext Markup Language, utilizando el software WebAcappella -Responsive Website Creator (Intuisphere, Francia 2016). El contenido se estructuró en los módulos: Historia, Trasplante, Enfermedades Autoinmunes, Links, Guías, Habla Equipo y Dudas. Los...
Background Takayasu's arteritis is a rare autoimmune vasculitis that affects medium and large vessels, especially the aorta and its main branches. Autologous stem cell transplantation for autoimmune disease has been shown to be effective in some refractory cases. Objectives Describe our experience with autologous hematopoietic stem cell transplantation (AHSCT) in Takayasu's arteritis. Methods Three patients with refractory Takayasu's arteritis were treated with AHSCT. Hematopoietic stem cells were mobilized with 2g/m2 cyclophosphamide plus G-CSF, and subsequently harvested by leukoapheresis and cryopreserved, nonmanipulated. Patients then received 200mg/kg IV cyclophosphamide and 4.5mg/kg rabbit anti-thymocyte globulin (ATG) over five consecutive days, followed by infusion of the cryopreserved cells. Results The procedure was well tolerated by all patients. Side effects included fever, rash, nausea, vomiting and neutropenic fever. None of the patients presented severe side effects during or after AHSCT. One patient presented CMV reactivation after transplant and was preemptively treated, without clinical manifestations of disease.Case 1: 41 year-old female, with a history of dizziness, claudication of upper and lower limbs, non-palpable right radial pulse and transient visual impairment for over 13 years. Arteriography showed irregularity and stenosis of abdominal aorta, right and left iliac arteries, left subclavian and carotid arteries. She had been unsuccessfully treated with steroids, methotrexate, cyclophosphamide, chlorambucil and mycophenolate mofetil. On follow-up of 127 months after AHSCT, she presented clinical and laboratory remission. Case 2: 30 year-old female, with intermittent visual deficits, upper limb claudication, subclavian steal syndrome and 3 episodes of transient ischemic strokes. Previous treatments included steroids, azathioprine, cyclophosphamide and mycophenolate mofetil, besides a stent placed in the right carotid artery. On 43 months follow-up, she remained symptomatic with high levels of acute phase reactants, indicating refractoriness to AHSCT. After transplant, she also failed etanercept injections, but disease activity was further controlled with tocilizumab. Case 3: 39 year-old male with intestinal claudication, renovascular hypertension, upper limb claudication and dizziness. Magnetic angioresonance (NMR) images evidenced right renal and mesenteric artery stenosis, and bilateral critical narrowing of carotid, subclavian, brachial and axilary arteries, and irregularities along the aorta. On 15 months follow-up, there was clinical and laboratorial improvement. Patients 1 and 3 presented partial reversal of vessel stenosis detected by NMR images. Conclusions Very few cases of vasculitides treated with AHSCT have been reported in the literature. To the best of our knowledge, this is the largest reported series of transplanted Takayasu's patients. Although response to transplant was not universal, beneficial effects were verified in two out of three patients, who remained in...
BACKGROUND Juvenile idiopathic arthritis (JIA) is a chronic inflammatory disease that develops until the age of 16. The involvement of the temporomandibular joint (TMJ) was reported in 17-87% of JIA patients. Although TMJ arthritis may be asymptomatic, it can lead to changes in facial growth. The panoramic radiograph (PR) of the mandible provides an overview of the teeth and bones of the face including the TMJ. Unlike other imaging exams, it is a non-expensive method with high availability and does not require sedation in young children.
BackgroundSystemic sclerosis (SSc) is associated with impairment of physical function, daily life activities and quality of life. Autologous hematopoietic stem cell transplantation (AHSCT) has been investigated as treatment for patients with severe SSc, promoting reduction of skin thickening and at least stabilization of pulmonary involvement.ObjectivesTo evaluate the impact of AHSCT on skin involvement, functional capacity and quality of life (QoL) of SSc patients, and to compare these results with those from SSc patients under conventional immunosuppressive treatment.MethodsThis is a prospective longitudinal study of a cohort of SSc patients treated at a University Hospital in Brazil. Patients treated with AHSCT (transplant group) and patients under conventional immunosuppressive treatment (control group) were evaluated before, 6 and 12 months after treatment. The evaluations included respiratory muscle strength test (maximal inspiratory pressure – MIP and maximal expiratory pressure - MEP), hand function assessments (hand-grip strength, finger-to-palm distance - FTP, COCHIN questionnaire), six-minute walk test (6MWT), modified Rodnan's skin score (mRSS) and quality of life questionnaire (SF-36). Statistical significance was established at p<0.05.ResultsNine SSc patients were included in the transplant group and 8 in the control group. When compared before, 6 and 12 months after AHSCT, patients presented improvement of MIP (p=0.0019), 6MWT distance (p=0.0459), right hand FTP (p=0.0086), right (p=0.0001) and left (p=0.0001) hand-grip strength, COCHIN (p=0.0005), mRSS (p=0.0007), and of the physical components score of SF-36 (p=0.0236). No significant differences were detected in the remaining evaluations. In the control group, no significant changes were observed 6 and 12 months after treatment at any of the evaluations. Patients from the transplant group presented better scores than those from the control group at the 6 months time point regarding MIP (p=0.0050), 6MWT distance (p=0.0418), right (p=0.0024) and left (p=0.0047) hand-grip strength, COCHIN (p=0.0088), mRSS (p=0.0272), and physical components score of SF-36 (p=0.0342). At the 12 months time point, transplanted patients presented better scores than controls concerning MIP (p=0.0242), right (p=0.0131) and left (p=0.0005) hand-grip strength, and physical components score of SF-36 (p=0.0350).ConclusionsAHSCT promotes improvement of functional capacity, skin involvement and of physical components of quality of life in SSc patients. Scores are also significantly better than those from patients treated with conventional immunosuppressive treatment. These results can be interpreted as positive outcomes of AHSCT for SSc.ReferencesJAMA 2014;311:2490–8.Lancet 2013;381:1116–24.Disclosure of InterestNone declared
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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