Hematopoietic stem cell transplantation for systemic sclerosis: Brazilian experience.

BACKGROUND: In the past 20 years, hematopoietic stem cell transplantation (HSCT) has been investigated as treatment for systemic sclerosis (SSc). The goal of HSCT is to eradicate the autoreactive immune system, which is replaced by a new immune repertoire with long-lasting regulation and tolerance to autoantigens. Here, we describe the clinical outcomes of severe and refractory SSc patients that underwent HSCT at a single Brazilian center. PATIENTS AND METHODS: This is a longitudinal and retrospective study, including 70 adult SSc patients, with an established diagnosis of SSc, and who underwent autologous HSCT from 2009 to 2016. The procedure included harvesting and cryopreservation of autologous hematopoietic progenitor cells, followed by administration of an immunoablative regimen and subsequent infusion of the previously collected cells. Patients were evaluated immediately before transplantation, at 6 months and then yearly until at least 5-years of post-transplantation follow-up. At each evaluation time point, patients underwent clinical examination, including modified Rodnan's skin score (mRSS) assessment, echocardiography, high-resolution computed tomography of the lungs and pulmonary function. RESULTS: Median (range) age was 35.9 (19-59), with 57 (81.4%) female and median (range) non-Raynaud's disease duration of 2 (1-7) years. Before transplantation, 96% of the patients had diffuse skin involvement, 84.2%, interstitial lung disease and 67%, positive anti-topoisomerase I antibodies. Skin involvement significantly improved, with a decline in mRSS at all post-transplantation time points until at least 5-years of follow-up. When patients with pre-HSCT interstitial lung disease were analyzed, there was an improvement in pulmonary function (forced vital capacity and diffusing capacity of lung for carbon monoxide) over the 5-year follow-up. Overall survival was 81% and progression-free survival was 70.5% at 8-years after HSCT. Three patients died due to transplant-related toxicity, 9 patients died over follow-up due to disease reactivation and one patient died due to thrombotic thrombocytopenic purpura. CONCLUSIONS: Autologous hematopoietic progenitor cell transplantation improves skin and interstitial lung involvement. These results are in line with the international experience and support HSCT as a viable therapeutic alternative for patients with severe and progressive systemic sclerosis.

Hematopoietic stem cell transplantation for systemic sclerosis: Brazilian experience

Abstract Background: In the past 20 years, hematopoietic stem cell transplantation (HSCT) has been investigated as treatment for systemic sclerosis (SSc). The goal of HSCT is to eradicate the autoreactive immune system, which is replaced by a new immune repertoire with long-lasting regulation and tolerance to autoantigens. Here, we describe the clinical outcomes of severe and refractory SSc patients that underwent HSCT at a single Brazilian center. Patients and methods: This is a longitudinal and retrospective study, including 70 adult SSc patients, with an established diagnosis of SSc, and who underwent autologous HSCT from 2009 to 2016. The procedure included harvesting and cryopreservation of autologous hematopoietic progenitor cells, followed by administration of an immunoablative regimen and subsequent infusion of the previously collected cells. Patients were evaluated immediately before transplantation, at 6 months and then yearly until at least 5-years of post-transplantation follow-up. At each evaluation time point, patients underwent clinical examination, including modified Rodnan's skin score (mRSS) assessment, echocardiography, high-resolution computed tomography of the lungs and pulmonary function. Results: Median (range) age was 35.9 (19-59), with 57 (81.4%) female and median (range) non-Raynaud's disease duration of 2 (1-7) years. Before transplantation, 96% of the patients had diffuse skin involvement, 84.2%, interstitial lung disease and 67%, positive anti-topoisomerase I antibodies. Skin involvement significantly improved, with a decline in mRSS at all post-transplantation time points until at least 5-years of follow-up. When patients with pre-HSCT interstitial lung disease were analyzed, there was an improvement in pulmonary function (forced vital capacity and diffusing capacity of lung for carbon monoxide) over the 5-year follow-up. Overall survival was 81% and progression-free survival was 70.5% at 8-years after HSCT. Three patients died due to transplant-related toxicity, 9 patients died over follow-up due to disease reactivation and one patient died due to thrombotic thrombocytopenic purpura. Conclusions: Autologous hematopoietic progenitor cell transplantation improves skin and interstitial lung involvement. These results are in line with the international experience and support HSCT as a viable therapeutic alternative for patients with severe and progressive systemic sclerosis.(AU)

Higher Anti-A/B isoagglutinin titers of IgG class, but not of IgM, are associated with increased red blood cell transfusion requirements in bone marrow transplantation with major ABO-mismatch.

BACKGROUND: Major ABO mismatch between donor and recipient in bone marrow transplantation (BMT) may cause hemolysis, delayed red blood cell (RBC) engraftment and pure red cell aplasia (PRCA), which result in increased transfusion needs. High pretransplant anti-A/B antibody titers have been associated with increased risk of PRCA. Herein, we studied the impact of anti-A/B titers on transfusion needs after BMT with major ABO mismatch. METHODS: We reviewed the medical charts of 27 patients who underwent to BMT with major ABO mismatch and categorized them into two groups according to anti-A/B titers of IgG (≤16 and ≥32). We recorded the number of RBC and platelet units transfused in the first 180 days after transplantation. We also evaluated the impact of anti-A/B titers on overall survival. RESULTS: Patients with anti-A/B titer ≥32 of IgG class required more RBC transfusion than patients with titer ≤16 (6.60±4.55 vs 21.29±14.68; P=.03). Anti-A/B of IgM class had no impact on both RBC and platelet transfusion needs. Anti-A/B titers had no impact on overall survival. CONCLUSION: Higher titers of anti-A/B antibodies of IgG class, but not of IgM, are associated with a higher demand for RBC transfusion.

Violência doméstica contra crianças: nível de conhecimento dos pais de crianças em escolas pública e privada / Domestic violence against children: knowledge of parents of children enrolled in public and private schools

Objetivo: Investigar o conhecimento dos pais ou responsáveis por crianças matriculadas no ensino fundamental sobre a violência infantil e identificar a sua postura em relação à educação de seus filhos. Métodos: Trata-se de um estudo descritivo. Os dados foram coletados a partir de um questionário, que abordava questões sobre violência infantil e práticas educativas, respondidopelos pais/responsáveis de alunos matriculados em uma escola pública e, em outra particular, em Catanduva, São Paulo. Resultados: Foram aplicados 1.429 questionários na escola pública e 256 na particular, havendo retorno de, respectivamente, 670 (47%) e 115 (45%) deles. As respostas foram (particular/pública): 1) não aceitam que educadores punam com castigos físicos (99/96%); 2) chamam atenção dos filhos diante de comportamento inadequado (90/92%); 3) discordam que ôpalmadaõ prepara a criança para a vida (80/66%); 4) acham incorreto repreender os filhos na frente de outros (87/73%); 5) concordam que ôtapas/palmadasõ prejudicam o relacionamento pais/filhos (59/57%); 6) concordam que conversar sobre ôerrosõ faz parte da educação (97/91%); 7) concordam que ôxingarõ causa danos à criança (81/60%); 8) acham que ômedoõ não significa ôrespeitoõ (86/57%); 9) concordam que ôcriança não esquece fácilõ(92/79%); 10) concordam com a notificação de violência infantil (93/90%); 11) preocupam-se com a violência na casa alheia (51/30%); 12) sabem que agressões domésticas podem levar à morte (97/94%); 13) consideram baixa a violência em seus lares (89/86%). Houve diferença significativa entre os grupos para as respostas às questões 3, 4, 7, 8, 9 e 11 (p≤0,001).Conclusões: Os entrevistados demonstraram conhecimento satisfatório sobre violência infantil, embora os da escola pública tenham apresentado pior desempenho nas questões relacionadas à educação familiar.Palavras-chave: violência doméstica; maus-tratos infantis;família; educação infantil.

Objective: To study the knowledge about domestic violence of parents and caregivers of children enrolled in elementary schools, and to identify how they deal with this subject in their children education.Methods: This descriptive study used a questionnaire about child abuse and educational practices applied to parents/caregivers of children from one public and one private school in Catanduva, São Paulo State, Brazil.Results: The questionnaires were sent to caregivers in the public school and (1429) and private school (256), and they were answered respectively by 670 (47%) and 115 (45%) of them. The answers were (private/public school): 1) do not approve physical punishment by educators (99/96%); 2) draw the attention of children to bad behavior(90/92%); 3) disagree that ôslappingõ prepares for life (80/66%); 4) agree that it is wrong to reprimand children in front of others (87/73%); 5) agree that ôslaps/smacksõ affect the relationship between parents and children (59/57%); 6) talk about ômistakesõ as a part of education (97/91%); 7) agree that ôinsultingõ causes damage to a child (81/60%); 8) think that ôfearõ is not the same as ôrespectõ (86/57%); 9) agree that a ôchild does not forget easilyõ (92/79%); 10) agree with child abuse notification (93/90%); 11) worry about violence in someone else's house (51/30%); 12) know that domestic violence may kill (97/94%); 13) consider the violence level in their homes low (89/86%). There were diferences between schools regarding questions 3, 4, 7, 8, 9 and 11 (p≤0.001).Conclusions: Caregivers of children attending elementary schools are aware of the main concepts regarding child abuse, although participants from the public school presented a worse performance regarding educational questions.