Clinical studies using stem cells for treatment of retinal diseases: state of the art / Estudos clínicos com uso de células-tronco para tratamento de doenças da retina: estado da arte

ABSTRACT Degenerative retinal diseases such as retinitis pigmentosa, Stargardt's macular dystrophy, and age-related macular degeneration are characterized by irreversible loss of vision due to direct or indirect photoreceptor damage. No effective treatments exist, but stem cell studies have shown promising results. Our aim with this review was to describe the types of stem cells that are under study, their effects, and the main clinical trials involving them.

RESUMO As doenças degenerativas da retina, como retinose pigmentar, distrofia macular de Stargardt e degeneração macular relaciona à idade, são caracterizadas por perda irre versível da visão devido a danos diretos ou indiretos aos fotorreceptores. Não existem tratamentos eficazes, porém os estudos com células-tronco mostraram resultados promissores. Nosso objetivo com esta revisão foi descrever os tipos de células-tronco em estudo, seus efeitos e os principais ensaios clínicos que as envolvem.

Clinical studies using stem cells for treatment of retinal diseases: state of the art.

Degenerative retinal diseases such as retinitis pigmentosa, Stargardt's macular dystrophy, and age-related macular degeneration are characterized by irreversible loss of vision due to direct or indirect photoreceptor damage. No effective treatments exist, but stem cell studies have shown promising results. Our aim with this review was to describe the types of stem cells that are under study, their effects, and the main clinical trials involving them.

Durometry as an alternative tool to the modified Rodnans skin score in the assessment of diffuse systemic sclerosis patients: a cross-sectional study

Abstract Background The reproducibility and reliability of the modified Rodnan's Skin Score (mRSS) are debated due to investigator-related subjectivity. Here, we evaluate if durometry correlates with mRSS in patients with diffuse systemic sclerosis (SSc). Methods This cross-sectional study was conducted from December 2018 to June 2019, including 58 diffuse SSc patients. Two certified researchers, blind to each other's scores, performed the mRSS, followed by durometry at 17 predefined skin sites. For durometry and mRSS, individual scores per skin site were registered. Durometry and mRSS results measured by each researcher, as well as scores from different researchers, were compared. Skin thickness measurements from forearm skin biopsies were available in a subset of the patients, for comparisons. Statistical analyses included Cohen's Kappa Coefficient, Intraclass Correlation Coefficient, Kendall's Coefficient and Spearman's test. Results Mean (standard deviation, SD) patient age was 44.8 (12.9) years, and 88% were female. Inter-rater agreement varied from 0.88 to 0.99 (Intraclass correlation coefficient) for durometry, and 0.54 to 0.79 (Cohen's Kappa coefficient) for mRSS, according to the specific evaluated sites. When data were compared with skin thickness assessed in forearm biopsies, durometry correlated better with skin thickness than mRSS. Conclusion Durometry may be considered as an alternative method to quantify skin involvement in patients with diffuse SSc. The strong inter-rater agreement suggests that the method may be useful for the assessment of patients by multiple researchers, as in clinical trials.(AU)

Curativo gel de clorexidina no transplante de células-tronco hematopoéticas / Vendaje gel de clorhexidina en el trasplante de células madre hematopoyéticas / Chlorhexidine gel dressing in hematopoietic stem cell transplantation

Resumo Objetivo Acompanhar o uso do curativo gel de clorexidina em cateter venoso central de crianças e adultos submetidos ao transplante de células-tronco hematopoéticas para verificar a ocorrência de infecção da corrente sanguínea relacionada ao cateter e irritação cutânea na vigência deste curativo. Métodos Estudo observacional e prospectivo, com 25 pacientes com cateter venoso central inserido para realização do transplante de células-tronco hematopoéticas. Diariamente avaliou-se as características do curativo, da pele, clínicas do paciente e relacionadas a infecção. Os pacientes foram acompanhados a partir do primeiro dia da inserção do CVC até a sua remoção, até o limite de 45 dias a partir da data de inserção do CVC. Resultados A infecção da corrente sanguínea relacionada ao cateter ocorreu em 28% dos pacientes, houve associação entre neutropenia febril e infecção (p<0,01). A irritação cutânea foi identificada em 24% dos pacientes. Não foi identificada associação entre infecção da corrente sanguínea relacionada ao cateter e a irritação cutânea (p=0,51). Houve associação significativa entre irritação cutânea e suspensão do uso do curativo (p=0,03). As trocas não programadas ocorreram em 50% das trocas identificadas e o principal motivo foi a presença de sangue no curativo (57,8%). Conclusão O uso do curativo gel de clorexidina em pacientes submetidos ao TCTH demonstrou ser uma medida eficaz na redução da ocorrência de infecções relacionadas ao cateter, quando comparado aos dados da literatura. Os casos de ICSRC encontrados foram associados, principalmente, à condição de neutropenia, muito comum nesta população. Os casos de irritação cutânea relacionados ao curativo identificados e a presença de sangue como principal motivo de troca não programada salientam a importância do conhecimento relacionado ao uso do curativo por parte dos enfermeiros assistenciais, a fim de criar protocolos que indiquem o uso e manejo do curativo com segurança.

Resumen Objetivo Hacer un seguimiento del uso del vendaje gel de clorhexidina en catéter venoso central (CVC) en niños y adultos sometidos al trasplante de células madre hematopoyéticas (TCMH) para verificar los casos de infección del flujo sanguíneo relacionados con el catéter (IFSRC) y la irritación cutánea durante la validez de este vendaje. Métodos Estudio observacional y prospectivo, con 25 pacientes con catéter venoso central insertado para realizar el trasplante de células madre hematopoyéticas. Diariamente se evaluaron las características del vendaje y de la piel, las características clínicas del paciente y las relacionadas con la infección. Los pacientes recibieron un seguimiento a partir del primer día de la inserción del CVC hasta su retirada, con un límite de 45 días a partir de la fecha de inserción del CVC. Resultados La infección del flujo sanguíneo relacionada con el catéter se presentó en el 28% de los pacientes, hubo relación entre neutropenia febril e infección (p<0,01). Se identificó irritación cutánea en el 24% de los pacientes. No se identificó relación entre la infección del flujo sanguíneo relacionada con el catéter y la irritación cutánea (p=0,51). Hubo relación significativa entre la irritación cutánea y la suspensión del uso del vendaje (p=0,03). Los cambios no programados sucedieron en el 50% de los cambios registrados, y el motivo principal fue la presencia de sangre en el vendaje (57,8%). Conclusión El uso del vendaje gel de clorhexidina en pacientes sometidos al TCMH demostró ser una medida eficaz para la reducción de casos de infecciones relacionadas con el catéter, en comparación con los datos de la literatura. Los casos de IFSRC encontrados se relacionaron principalmente con la condición de neutropenia, muy común en esta población. Los casos identificados de irritación cutánea relacionados con el vendaje y la presencia de sangre como principal motivo de cambio no programado resaltan la importancia del conocimiento del uso del vendaje por parte de los enfermeros asistenciales, a fin de crear protocolos que indiquen el uso y manejo seguro del vendaje.

Abstract Objective Monitor the use of chlorhexidine gel dressing in the central venous catheter of children and adults submitted to hematopoietic stem cell transplantation (HSCT) to check for catheter-related bloodstream infection and skin irritation while using this dressing. Methods This is a prospective observational study with 25 patients with central venous catheters (CVC) inserted for hematopoietic stem cell transplantation. Dressing characteristics, skin characteristics, clinical conditions of patients and infection-related characteristics were evaluated daily. Patients were monitored from the first day of CVC insertion until removal, up to 45 days from the CVC insertion date. Results Catheter-related bloodstream infection (CRBSI) occurred in 28% of all patients, with an association between febrile neutropenia and infection (p<0.01). Skin irritation was identified in 24% of patients. No association was found between catheter-related bloodstream infection and skin irritation (p=0.51). A significant association was observed between skin irritation and dressing removal (p=0.03). Unscheduled dressing changes corresponded to 50% of all dressing changes identified during the study, and the main reason was presence of blood in the dressing (57.8%). Conclusion Using chlorhexidine gel dressing in patients submitted to HSCT proved to be an effective measure to reduce the occurrence of catheter-related infections, when compared to literature data. The cases of CRBSI found in this study were mainly associated with the condition of neutropenia, which is very common in this population. The cases of dressing-related skin irritation and presence of blood as the main reason for unscheduled change highlight the importance of nurses having proper knowledge about how to use this dressing in order to create protocols for safe dressing use and handling.

Consenso brasileiro para transplante de células-tronco hematopoéticas para tratamento de doenças autoimunes / Brazilian consensus on hematopoietic stem cell transplantation for autoimmune diseases

Neste trabalho, foram revisadas a literatura internacional e a experiência nacional com transplante de células-tronco hematopoéticas (TCTH) para doenças autoimunes. A evidência acumulada indica que o TCTH autólogo pode beneficiar pacientes com esclerose múltipla em fase inflamatória, refratária aos tratamentos medicamentosos disponíveis, e pacientes com esclerose sistêmica cutânea difusa de caráter progressivo, com ou sem comprometimento sistêmico. Esse tratamento deveria ser disponibilizado na rede pública de saúde, numa fase inicial, em centros de referência com experiência em TCTH e no manejo clínico de doenças autoimunes sistêmicas graves.

In this paper, international literature and national experience on hematopoietic stem cell transplantation (HSCT) for autoimmune diseases were reviewed. Cumulative evidence indicates that autologous HSCT may benefit patients with inflammatory multiple esclerosis, refractory to available drug therapy, and progressive forms of diffuse cutaneous systemic sclerosis with or without systemic involvement. Initially, this treatment should be available in reference centers of the public health system, with experience in performing HSCT and in treating severe systemic autoimmune diseases.