METHODOLOGY: A prospective comparison of subjective global assessment (SGA), and anthropometry (mid-arm muscle circumference, MAMC; triceps skinfold thickness, TST) between elderly stroke patients on long-term NG feeding and matched controls was performed. Selected clinicians and carers of patients were interviewed to assess their knowledge and attitudes to gastrostomy feeding.
RESULTS: 140 patients (70 NG, 70 oral) were recruited between September 2010 and February 2011. Nutritional status was poorer in the NG compared to the oral group (SGA grade C 38.6% NG vs 0% oral, p<0.001; TST males 10.7 + 3.7 mm NG vs 15.4 + 4.6 mm oral, p<0.001; MAMCmales 187.9 + 40.4 mm NG vs 228.7 + 31.8 mm oral, p<0.001). 45 (64.3%) patients on long-term NG feeding reported complications, mainly consisting of dislodgement (50.5%), aspiration of feed content (8.6%) and trauma from insertion (4.3%). Among 20 clinicians from relevant speciliaties who were interviewed, only 11 (55%) clinicians would routinely recommend a PEG. All neurologists (100%) would recommend a PEG, whilst the response was mixed among non-neurologists. Among carers, lack of information (47.1%) was the commonest reason stated for not choosing a PEG.
CONCLUSION: Elderly patients with stroke on long term NG feeding have a poor nutritional status. Lack of recommendation by clinicians appears to be a major barrier to PEG feeding in these patients.
METHODS: In this randomized, double-blind, phase 3 trial, we assigned, in a 2:1 ratio, adults with transfusion-dependent β-thalassemia to receive best supportive care plus luspatercept (at a dose of 1.00 to 1.25 mg per kilogram of body weight) or placebo for at least 48 weeks. The primary end point was the percentage of patients who had a reduction in the transfusion burden of at least 33% from baseline during weeks 13 through 24 plus a reduction of at least 2 red-cell units over this 12-week interval. Other efficacy end points included reductions in the transfusion burden during any 12-week interval and results of iron studies.
RESULTS: A total of 224 patients were assigned to the luspatercept group and 112 to the placebo group. Luspatercept or placebo was administered for a median of approximately 64 weeks in both groups. The percentage of patients who had a reduction in the transfusion burden of at least 33% from baseline during weeks 13 through 24 plus a reduction of at least 2 red-cell units over this 12-week interval was significantly greater in the luspatercept group than in the placebo group (21.4% vs. 4.5%, P<0.001). During any 12-week interval, the percentage of patients who had a reduction in transfusion burden of at least 33% was greater in the luspatercept group than in the placebo group (70.5% vs. 29.5%), as was the percentage of those who had a reduction of at least 50% (40.2% vs. 6.3%). The least-squares mean difference between the groups in serum ferritin levels at week 48 was -348 μg per liter (95% confidence interval, -517 to -179) in favor of luspatercept. Adverse events of transient bone pain, arthralgia, dizziness, hypertension, and hyperuricemia were more common with luspatercept than placebo.
CONCLUSIONS: The percentage of patients with transfusion-dependent β-thalassemia who had a reduction in transfusion burden was significantly greater in the luspatercept group than in the placebo group, and few adverse events led to the discontinuation of treatment. (Funded by Celgene and Acceleron Pharma; BELIEVE ClinicalTrials.gov number, NCT02604433; EudraCT number, 2015-003224-31.).