Pancreatic insufficient children with cystic fibrosis (CF) are routinely supplemented with fat-soluble vitamins A, D, E, and K, and serum vitamin levels are checked in annual review assessments. Prior to 2018, the formulations involved preparations that provide Vitamin E, and K separately, but A and D together. This has proven to be challenging for parents and carers looking after young children with CF. Anew formulation (Paravit – CF), containing all the required vitamins in one easily deliverable format, became available, but uncertainty remained as to their efficacy in maintaining healthy vitamin levels. This study, temporally explored the efficacy of this approach, comparing data from before and after supplementation. Royal Manchester Children's Hospital (RMCH), a large tertiary CF centre in northwest England, cares for 330 children with CF, through direct input (200 children) and by shared care arrangements (130 children). For children under direct care at RMCH, an electronic registry (THRIVE) captures anonymised pre- and post-supplementation data, including vitamin levels and anthropometric parameters. This study temporally explored serum vitamin levels and growth parameters (weight, height, and BMI) before and after Paravit – CF supplementation. In this preliminary analysis, complete data was available on only 78 children. Vitamin A levels were slightly higher (p < 0.001), and vitamin E levels were slightly lower (p = 0.027), in the post-supplementation group. No significant change was observed in the vitamin D levels. Furthermore, anthropometric parameters remained stable. A new vitamin supplementation program using a single formulation did not result in a loss of efficacy in attaining desirable vitamin levels and in maintaining anthropometric parameters. Future research should explore parental satisfaction using patient-reported outcome measures.

CFTR modulators, improve lung function, body mass index, and reduce pulmonary exacerbations. There is some evidence from clinical trials1Rosenfeld et al.ARRIVAL.Lancet Resp Med. 2018 Jul; 6PubMed Google Scholar that CFTR modulators may restore some degree of pancreatic function, but this has not been thoroughly investigated in the "real world." A number of children from our centre's cohort, a large tertiary paediatric hospital in Northwest England, have reported a reduction in the symptoms of pancreatic insufficiency since commencing modulator therapy. We aim to investigate the effect of modulator therapy on pancreatic function by repeating faecal elastase (FE) sampling on children reporting improved symptoms. FE samples were repeated from children with clinical evidence of improved pancreatic function. Clinical symptoms were lack of malabsorption symptoms (loose and/or oily stools) when Creon doses were not given with food. Often with a change in bowel habit with greater tendency toward constipation (not resulting from change in dietary fibre and/or fluids). To date 8 children aged 2–14 yrs., with at least one delF508 mutation, have had a FE repeated. 3 patients had an improvement to over 200 ug/g with a FE at birth of <15 ug/g in all cases. 2 had an improvement to between 15–200 ug/g. 3 remained insufficient at <15 ug/g. Some patients on CFTR modulators demonstrate recovery to pancreatic sufficient levels, and others partial recovery. However, improvement of clinical symptoms of malabsorption does not always correlate with biochemical analysis, which can make advice/dosing of pancreatic replacement therapy challenging. Data collection is ongoing, with further data becoming available. Our data will have a direct impact on clinical practice and could change annual review screening for pancreatic sufficiency in all CF pancreatic insufficient patients.

Background: Pancreatic-insufficient children with cystic fibrosis (CF) are routinely supplemented with fat-soluble vitamins A, D, E, and K, according to national guidelines, and serum vitamin levels are checked in annual review assessments.Traditionally, the formulations have involved preparations that provide vitamins E and K separately and vitamins A and D together.This has proven to be challenging to parents and caregivers looking after young children with CF.A new formulation (Paravit-CF, Parapharm Development Ltd, Reading, UK) containing all the required vitamins in one easily deliverable format became available in the United Kingdom in 2018, but uncertainty remained as to its efficacy in maintaining healthy vitamin levels.This study explored the efficacy of this approach, comparing data from before and after.Methods: Royal Manchester Children's Hospital (RMCH), is a large tertiary CF center in northwest England that cares for 350 children with CF through direct input (200 children) and shared care arrangements (150 children).An electronic registry (THRIVE) captures anonymized pre-and postsupplementation data, including vitamin levels, anthropometric parameters, and patient-reported outcome measures, for children under direct care at RMCH.This study explores serum vitamin levels and growth parameters (weight, height, BMI) before and after Paravit-CF supplementation.Results: In this preliminary analysis, complete data were available for 45 children.No significant change was observed in vitamin A, D, and E levels before and after the Paravit-CF formulation was introduced0 and, anthropometric parameters remained stable.Conclusions: A new vitamin supplementation program using a single formulation did not result in a loss of efficacy in attaining desirable vitamin levels and in maintaining anthropometric parameters.Subsequent research should explore parental satisfaction using this approach using patient-reported outcome measures.

Age at CFLD diagnosis, years (SD) Splenomegaly at diagnosis of CFLD Diabetes Meconium ileus Death Progression (n = 80) 7.3 (3.5) 61 (76%) 32 (40%) 21 (26%) 26 (33%) No progression (n = 133) 8.9 (4.0) 42 (32%) 36 (27%) 34 (26%) 10 (8%) p 0.004 <0.001 0.05 0.91 <0.001 [Characteristics between patients with and without progression of CFLDv] Conclusion: Splenomegaly was the only significant marker of CFLD which was present in children who had progressive liver disease, indicating established liver disease at the time of diagnosis.There was no clear evidence that UDCA reduced the rate of progression of liver disease in those with established liver disease, but may do so if given prior to the onset of CFLD.