Motor function and safety after allogeneic cord blood and cord tissue-derived mesenchymal stromal cells in cerebral palsy: An open-label, randomized trial.

AIM: To evaluate safety and motor function after treatment with allogeneic umbilical cord blood (AlloCB) or umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in children with cerebral palsy (CP). METHOD: Ninety-one children (52 males, 39 females; median age 3 years 7 months [range 2-5 years]) with CP due to hypoxic-ischemic encephalopathy, stroke, or periventricular leukomalacia were randomized to three arms: (1) the AlloCB group received 10 × 107 AlloCB total nucleated cells (TNC) per kilogram at baseline (n = 31); (2) the hCT-MSC group received 2 × 106 hCT-MSC at baseline, 3 months, and 6 months (n = 28); (3) the natural history control group received 10 × 107 AlloCB TNC per kilogram at 12 months (n = 31). Motor function was assessed with the Gross Motor Function Measure-66 (GMFM-66) and Peabody Developmental Motor Scale, Second Edition. RESULTS: Infusions (n = 143) were well tolerated, with eight infusion reactions (three in the AlloCB group, five in hCT-MSC) and no other safety concerns. At 12 months, the mean differences (95% confidence intervals [CI]) between actual and expected changes in GMFM-66 score were AlloCB 5.8 points (3.4-8.2), hCT-MSC 4.3 (2.2-6.4), and natural history 3.1 (1.4-5.0). In exploratory, post hoc analysis, the mean GMFM-66 score (95% CI) of the hCT-MSC group was 1.4 points higher than natural history (-1.1 to 4.0; p = 0.27), and the AlloCB group was 3.3 points higher than natural history (0.59-5.93; p = 0.02) after adjustment for baseline Gross Motor Function Classification System level, GMFM-66 score, and etiology. INTERPRETATION: High-dose AlloCB is a potential cell therapy for CP and should be further tested in a randomized, blinded, placebo-controlled trial. WHAT THIS PAPER ADDS: Unrelated donor allogeneic umbilical cord blood (AlloCB) and human umbilical cord tissue-derived mesenchymal stromal cell infusion is safe in young children with cerebral palsy. Significant changes in motor function were not observed 6 months after treatment. One year later, treatment with AlloCB was associated with greater increases in Gross Motor Function Measure-66 scores.

Aboriginal people's perceptions of patient-reported outcome measures in the assessment of diabetes health-related quality of life.

BACKGROUND: Patient-reported outcome measures (PROMs) provide clinicians and consumers a platform to inform and improve healthcare planning and management. Aboriginal people experience disproportionately high rates of chronic diseases, including type 2 diabetes. Treatment and management require holistic approaches that draw on culturally relevant resources and assessment tools. This study explored perceptions of Aboriginal people about two diabetes management-related PROMs (PROMIS-29, PAID Scale). METHODS: Twenty-nine Aboriginal people living with diabetes in the Shoalhaven discussed two PROMs in one of four focus groups or at an individual interview. Preliminary data coding was conducted by clinician researchers, with thematic analysis overseen by Aboriginal co-researchers. Subsequent individual interviews with participants were undertaken to seek further feedback and articulate what is needed to improve methods of evaluating Aboriginal people's self-reported quality of life and diabetes management. RESULTS: The PROMs did not capture information or knowledge that Aboriginal people considered relevant to their diabetes-related health care. Participants' recommendations included adapting survey materials to be more culturally sensitive; for example, by improving the alignment of measures with common day-to-day activities. This study also describes a genuine collaborative, Aboriginal community-guided approach to evaluate 'fit-for-purpose' diabetes management tools. CONCLUSIONS: Appropriate evaluation methods are paramount to address the disproportionate burden of diabetes experienced by Aboriginal peoples and overcome inverse diabetes care. Our learnings will contribute to development of tools, resources or methods that capture culturally tailored outcome measures. Study findings are relevant to clinicians and researchers using and/or developing Patient Reported Measures, particularly in relation to the practicality of tools for First Nations peoples.

Assessing cultural appropriateness of patient-reported outcome measures for Aboriginal people with diabetes: study protocol.

BACKGROUND: Patient-reported outcome measures (PROMs) provide patients with a platform to report on healthcare services and interventions, including the management of diabetes and other chronic diseases. Given the major disparity in incidence and prevalence of diabetes between Aboriginal and non-Aboriginal people, it is crucial that PROMs meet the cultural, spiritual and medical needs of this high-risk population. Anecdotal evidence indicates that PROMs do not capture accurate information about the healthcare of Aboriginal people with Type 2 diabetes. Therefore, the aim of this study is to explore the perceptions of Aboriginal people in regional New South Wales (NSW), Australia, about the cultural appropriateness of a set of PROMs relating to diabetes management. METHODS/DESIGN: Up to 50 Aboriginal people aged 18 years or older with type 1 or type 2 diabetes living in the Shoalhaven region in NSW, will be recruited. Eligible, consenting participants will attend a focus group or individual interview where they will review the PROM tools (PROMIS 29 and PAID scale) currently used for diabetes management. Semi-structured questions will be used to gather information about PROMs and discussions will be recorded for analysis. Research team members will yarn about the data with Aboriginal co-researchers and community stakeholders to apply an Indigenous lens to coding and thematic analysis. CONCLUSION: This study will explore the cultural appropriateness of government-initiated evaluation of health outcomes, and make recommendations about how these evaluations can be better suited to Aboriginal people. The lessons from this study will be of interest to those conducting research or quality assessment activities related to PROMs, and those involved with Aboriginal-focused research and project implementation.