RESUMEN
Favourable outcomes with CPX-351 versus conventional 7 + 3 were demonstrated in the pivotal phase III trial in adults aged 60-75 years with newly diagnosed, highrisk/secondary acute myeloid leukaemia (AML). As a complement to the clinical trial and to address important data gaps, the CPX-351 Real-World Effectiveness and SafeTy (CREST-UK; NCT05169307) study evaluated the use of CPX-351 in routine clinical practice in the UK, in 147 patients with newly diagnosed therapy-related AML or AML with myelodysplasia-related changes. Best response of complete remission or complete remission with incomplete platelet or neutrophil recovery was achieved by 53% of evaluable patients. Kaplan-Meier median overall survival (OS) was 12.8 months (95% confidence interval 9.2-15.3). Fifty (34%) patients proceeded to haematopoietic cell transplantation (HCT); median OS landmarked from the HCT date was not reached. There were no new safety concerns with CPX-351 identified in CREST-UK. Patients treated with CPX-351 in the outpatient setting spent an average of 24.4, 16.7, 28.2, and 27.7 fewer days on the ward compared with inpatients during first induction, second induction, first consolidation, and second consolidation, respectively. The results from CREST-UK provide valuable insights into the effectiveness, safety, and outpatient delivery of CPX-351 in routine clinical practice in the UK.
RESUMEN
OBJECTIVE: To determine the reliability of anthropometric and body composition measurements in children with special needs. DESIGN: Observational study. SETTING: Specialist support schools (primary and secondary) in Manchester, UK. PARTICIPANTS: 53 children with moderate-to-severe learning disability; 30 non-standers (14 boys) and 23 standers (15 boys). Mean ages were 11 years (range 3-20) for non-standers and 12.4 years (range 8-19) for standers. MEASURES: Anthropometric measures included: height/length, segmental measures, weight, skinfolds, body circumferences and body composition estimated from bioelectrical impedance analysis (BIA). These were measured twice, 2-4 weeks apart. MAIN OUTCOME MEASURES: Reliability was assessed using the technical error of measurement (TEM). RESULTS: The TEM for height and supine length was 0.55 cm for standers and 2.47 cm for non-standers, respectively. For non-standers, the TEMs for knee height and tibial length were 0.81 and 1.57 cm, respectively. The TEM for weight was 0.55 kg for standers and 0.75 kg for non-standers. For skinfold thickness, the TEM was smaller for non-standers than standers. The TEM for mid-upper arm circumference for standers and non-standers was 0.91 and 0.82 cm, respectively. The TEM for BIA in standers and non-standers was 34.7 and 54.1 Ω, respectively. Some measurements, including waist circumferences, were difficult to obtain reliably. CONCLUSIONS: Anthropometric and body composition measurements were feasible to obtain in children with special needs. However, the reliability of these measures differs between non-standers and standers and should be considered when choosing appropriate measures.
Asunto(s)
Antropometría/métodos , Composición Corporal/fisiología , Discapacidades para el Aprendizaje/fisiopatología , Adolescente , Estatura/fisiología , Peso Corporal/fisiología , Niño , Preescolar , Estudios de Factibilidad , Femenino , Humanos , Masculino , Reproducibilidad de los Resultados , Grosor de los Pliegues Cutáneos , Adulto JovenRESUMEN
AIM: To assess the vitamin D status of gastrostomy-fed children. METHODS: Vitamin D status was measured in 32 children aged five to 16 years recruited from special schools in Manchester, UK (53° 48' N). All children were receiving a nutritionally complete, commercially prepared enteral feed via gastrostomy, and had been established on this regimen for over 12 months. Serum concentrations of 25-hydroxyvitamin D (25OHD) were measured at the end of winter. Children with serum concentrations of 25OHD >50 nmol/L were considered to be sufficient, and those with concentrations <25 nmol/L were considered to be deficient. RESULTS: Approximately 83% of subjects had sufficient concentrations of serum 25OHD (>50 nmol/L). One subject was vitamin D deficient (serum 25OHD <25 nmol/L), and four were vitamin D insufficient (serum 25OHD >25 nmol/L - <50 nmol/L). The median vitamin D derived from enteral feeds was 9.45 µg/day; range 3.5-30; 13 children (41%) received less than 10 µg of vitamin D per day from their enteral feed. CONCLUSION: Nutritionally complete gastrostomy feeds may be protective against vitamin D deficiency in the majority of children with special needs. We recommend that all children over 1 year of age receive 10 µg (400 IU) of vitamin D, as recommended by the Scientific Advisory Committee on Nutrition (SACN).