A COST-EFFECTIVENESS ANALYSIS OF rhDNase IN CHILDREN WITH CYSTIC FIBROSIS

Objectives: This study compared the relative cost-effectiveness of daily recombinant human deoxyribonuclease (rhDNase), with alternate day rhDNase and hypertonic saline (HS) for treating children with cystic fibrosis (CF).Methods: A randomized controlled trial with a crossover design allocated 40 CF children consecutively to 12 weeks of daily rhDNase, alternate day rhDNase, or HS. The primary outcome measure was forced expiratory volume in 1 second (FEV1), a measure of lung function. All health resource use was prospectively documented for each patient and multiplied by unit costs to give a total health service cost for each 12-week treatment period. The nonparametric bootstrap method was used to present cost-effectiveness acceptability curves and net benefit statistics for each treatment comparison, for various hypothetical levels of the decision maker's ceiling ratio.Results: Compared with HS, there was a 14% improvement in FEV1 for daily rhDNase (95% CI, 5% to 23%), and a 12% improvement (95% CI, 2% to 22%) for alternate day rhDNase. For a ceiling ratio of £200 per 1% gain in FEV1, the mean net benefits of daily and alternate day rhDNase compared with HS were £1,158 (95% CI, −£621 to 2,842) and £1,188 (95% CI, −847 to 3,343), respectively; the mean net benefit of daily compared with alternate day rhDNase was *minus;£30 (95% CI, −£2,091 to 1,576).Conclusions: If decision makers are prepared to pay £200 for a 1% gain in FEV1 over a 12-week period, then on average either rhDNase strategy is cost-effective.

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Study of progesterone for the prevention of preterm birth in twins (STOPPIT): Findings from a trial-based cost-effectiveness analysis

International Journal of Technology Assessment in Health Care ◽

10.1017/s0266462310000036 ◽

2010 ◽

Vol 26 (2) ◽

pp. 141-148 ◽

Cited By ~ 9

Author(s):

Oya Eddama ◽

Stavros Petrou ◽

Dean Regier ◽

John Norrie ◽

Graeme MacLennan ◽

...

Keyword(s):

Economic Evaluation ◽

Preterm Birth ◽

Cost Effectiveness ◽

Controlled Trial ◽

Bootstrap Method ◽

Cost Difference ◽

Nonparametric Bootstrap ◽

Vaginal Progesterone ◽

Maternity Hospitals ◽

Twin Pregnancies

Objectives: Preterm birth contributes to a range of healthcare problems amongst infants surmounting to sizeable healthcare costs. Twin pregnancies are at particular risk of preterm birth. The objective of this study was to assess the cost-effectiveness of vaginal progesterone gel for the prevention of preterm birth in twin pregnancies.Methods: An economic evaluation was conducted alongside a randomized placebo controlled trial (the STOPPIT trial) of vaginal progesterone gel for the prevention of preterm birth in twin pregnancies. Five hundred women were recruited from nine maternity hospitals in the United Kingdom. The outcomes of the economic evaluation were presented in terms of net benefit statistics, cost-effectiveness acceptability curves, generated using the nonparametric bootstrap method, and the expected value of perfect information.Results: Mean health service costs between the period of randomization and discharge for mother and infant were £28,031 in the progesterone group and £25,972 in the placebo group, generating a mean nonsignificant cost difference of £2,059 (bootstrap mean cost difference £2,334; 95 percent confidence interval: −£5,023, £9,142; p = .33). The probability of progesterone being cost-effective was 20 percent at a willingness to pay threshold of £30,000 per preterm birth prevented. There is little economic justification for conducting further research into the use of vaginal progesterone gel in twin pregnancies for the prevention of preterm birth.Conclusions: Further studies of preventive interventions for preterm birth more generally are required given the scale of the clinical and economic burden of this condition. These studies should be sufficiently powered for economic endpoints and extend beyond hospital discharge.

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Protocol for the evaluation of cost-effectiveness and health equity impact of a school-based tobacco prevention programme in a cluster randomised controlled trial (the TOPAS study)

BMJ Open ◽

10.1136/bmjopen-2020-045476 ◽

2021 ◽

Vol 11 (8) ◽

pp. e045476

Author(s):

Anni-Maria Pulkki-Brännström ◽

Maria R Galanti ◽

Maria Nilsson

Keyword(s):

Cost Effectiveness ◽

Health Equity ◽

Tobacco Use ◽

Controlled Trial ◽

Health Agency ◽

Sensitivity Analyses ◽

Tobacco Prevention ◽

Net Benefit ◽

School Based ◽

Registration Number

IntroductionDespite a long-term downward trend in smoking prevalence, tobacco remains the number one risk factor for death and disability in Sweden. Globally, tobacco use generates a substantial economic burden for health systems and is also a major driver of socioeconomic inequalities in health. This article describes the planned cost-effectiveness and health equity impact evaluation of a multicomponent school-based programme to prevent the onset of tobacco use in adolescents.Methods and analysisCost-effectiveness of the multicomponent Tobacco-Free Duo programme will be evaluated against the educational component of the same programme only. An incremental cost-effectiveness ratio (ICER) will be calculated in terms of the cost per case prevented using the trial primary outcome and within-trial payer costs. If the ICER is negative, an incremental net benefit ratio will be calculated. Robustness of the results will be assessed through one-way sensitivity analyses. The slope index of inequality will be computed to assess the potential impact of the Tobacco-free Duo programme on education-related inequalities in the onset of smoking and in adult smoking cessation, comparing the two trial arms.Ethics and disseminationEthical approval was obtained from the Regional Ethics Review Board, Umeå (registration number 2017/255-31). The Public Health Agency of Sweden commissioned the study. The findings will be disseminated internationally within academia and to national and local policy-makers.Trial registration numberISRCTN52858080; Pre-results.

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Cost-effectiveness of habit-based advice for weight control versus usual care in general practice in the Ten Top Tips (10TT) trial: economic evaluation based on a randomised controlled trial

BMJ Open ◽

10.1136/bmjopen-2017-017511 ◽

2018 ◽

Vol 8 (8) ◽

pp. e017511 ◽

Cited By ~ 1

Author(s):

Nishma Patel ◽

Rebecca J Beeken ◽

Baptiste Leurent ◽

Rumana Z Omar ◽

Irwin Nazareth ◽

...

Keyword(s):

General Practice ◽

Economic Evaluation ◽

Cost Effectiveness ◽

Randomised Controlled Trial ◽

Health Service ◽

Usual Care ◽

Controlled Trial ◽

Cost Effective ◽

The Mean ◽

Randomised Controlled

ObjectiveTen Top Tips (10TT) is a primary care-led behavioural intervention which aims to help adults reduce and manage their weight by following 10 weight loss tips. The intervention promotes habit formation to encourage long-term behavioural changes. The aim of this study was to estimate the cost-effectiveness of 10TT in general practice from the perspective of the UK National Health Service.DesignAn economic evaluation was conducted alongside an individually randomised controlled trial.Setting14 general practitioner practices in England.ParticipantsAll patients were aged ≥18 years, with body mass index ≥30 kg/m2. A total of 537 patients were recruited; 270 received the usual care offered by their practices and 267 received the 10TT intervention.Outcomes measuresHealth service use and quality-adjusted life years (QALYs) were measured over 2 years. Analysis was conducted in terms of incremental net monetary benefits (NMBs), using non-parametric bootstrapping and multiple imputation.ResultsOver a 2-year time horizon, the mean costs and QALYs per patient in the 10TT group were £1889 (95% CI £1522 to £2566) and 1.51 (95% CI 1.44 to 1.58). The mean costs and QALYs for usual care were £1925 (95% CI £1599 to £2251) and 1.51 (95% CI 1.45 to 1.57), respectively. This generated a mean cost difference of −£36 (95% CI −£512 to £441) and a mean QALY difference of 0.001 (95% CI −0.080 to 0.082). The incremental NMB for 10TT versus usual care was £49 (95% CI −£1709 to £1800) at a maximum willingness to pay for a QALY of £20 000. 10TT had a 52% probability of being cost-effective at this threshold.ConclusionsCosts and QALYs for 10TT were not significantly different from usual care and therefore 10TT is as cost-effective as usual care. There was no evidence to recommend nor advice against offering 10TT to obese patients in general practices based on cost-effectiveness considerations.Trial registration numberISRCTN16347068; Post-results.

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Cost-effectiveness of computerised cognitive-behavioural therapy for anxiety and depression in primary care: Randomised controlled trial

The British Journal of Psychiatry ◽

10.1192/bjp.185.1.55 ◽

2004 ◽

Vol 185 (1) ◽

pp. 55-62 ◽

Cited By ~ 203

Author(s):

Paul McCrone ◽

Martin Knapp ◽

Judith Proudfoot ◽

Clash Ryden ◽

Kate Cavanagh ◽

...

Keyword(s):

Primary Care ◽

Cost Effectiveness ◽

Cognitive Behavioural Therapy ◽

Controlled Trial ◽

Cost Effective ◽

Behavioural Therapy ◽

Quality Adjusted Life Year ◽

Anxiety And Depression ◽

Net Benefit ◽

Cognitive Behavioural

BackgroundCognitive-behavioural therapy (CBT) is effective for treating anxiety and depression in primary care, but there is a shortage of therapists. Computer-delivered treatment may be a viable alternative.AimsTo assess the cost-effectiveness of computer-delivered CBT.MethodA sample of people with depression or anxiety were randomised to usual care (n= 128) or computer-delivered CBT (n= 146). Costs were available for 123 and 138 participants, respectively. Costs and depression scores were combined using the net benefit approach.ResultsService costs were £40 (90% CI-£28 to £148) higher over 8 months for computer-delivered CBT. Lost-employment costs were £407 (90% CI £196 to £586) less for this group. Valuing a 1-unit improvement on the Beck Depression Inventory at £40, there is an 81% chance that computer-delivered CBT is cost-effective, and it revealed a highly competitive cost per quality-adjusted life year.ConclusionsComputer-delivered CBT has a high probability of being cost-effective, even if a modest value is placed on unit improvements in depression.

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Is FEV1 associated with quality of life in children with cystic fibrosis? An original article

Immunopathologia Persa ◽

10.15171/ipp.2020.06 ◽

2019 ◽

Vol 6 (1) ◽

pp. e06-e06

Author(s):

Rohola Shirzadi ◽

Safoura Navaei ◽

Mohammadreza Modaresi ◽

Farzad Masiha

Keyword(s):

Quality Of Life ◽

Cystic Fibrosis ◽

Pulmonary Function ◽

Medical Center ◽

Cross Sectional Study ◽

Forced Expiratory Volume ◽

Psychological Status ◽

Cross Sectional ◽

The Mean

Introduction: Cystic fibrosis (CF) is a serious genetic life-shortening disease. Quality of life (QoL) measurement related to CF children is a relatively new field of research, which includes the patient’s perspective in research and clinical practice. Objectives: This study aimed to evaluate the QoL in children with CF and its association with FEV1 (forced expiratory volume in 1 second). Patients and Methods: This cross-sectional study was carried out on 7-14 years old children with CF attending children’s medical center from March 2017 to March 2018. Throat swab cultures and spirometry evaluation was performed for all patients. FEV1 was determined and the 6-min walk test (6MWT) was conducted. The patient’s psychological status was assessed using the Persian version of pediatric QoL inventory. ANOVA, t test, and chisquare tests were used for data analysis. Results: Seventy-six subjects with the mean age of 10.49±3.18 years were studied since 59% of them (n=44) were boys. The mean total QoL was 65.34±17.73. Patients with lower pulmonary function had a lower QoL. There was a significant association between FEV1 and school and emotional functioning (P=0.005 and P=0.002, respectively). A significant association was found between SPO2 (peripheral capillary oxygen saturation) reduction after 6MWT and FEV1 decline (P=0.001). Additionally, a significant association was detected between FEV1 and the distance walked during 6MWT (P=0.030). Conclusion: Regarding the association between pulmonary function and QoL in CF patients and lower QoL score in our study, the importance of assessing pulmonary function in these patients should not be neglected.

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Sputum and plasma adiponectin levels in clinically stable adult cystic fibrosis patients with CFTR I1234V mutation

10.21203/rs.2.19105/v1 ◽

2019 ◽

Author(s):

Atqah AbdulWahab ◽

Mona Allangawi ◽

Merlin Thomas ◽

Ilham Bettahi ◽

Siveen K. Sivaraman ◽

...

Keyword(s):

Cystic Fibrosis ◽

Adiponectin Level ◽

Pancreatic Insufficiency ◽

Forced Expiratory Volume ◽

Healthy Controls ◽

Cross Sectional ◽

Plasma Adiponectin ◽

Inflammatory Status ◽

Significant Difference ◽

The Mean

Abstract Background: Cystic fibrosis (CF) lung disease is associated with chronic inflammation leading to progress in lung function. Adiponectin is a predominantly anti-inflammatory adipokine that may have a role in CF lung. This study aims to determine total sputum and total plasma adiponectin levels in clinically stable adults CF patients with CFTR I1234V mutation, compared to plasma adiponectin levels in healthy controls and to investigate their correlations with body mass index (BMI) and spirometry in patients with CF. Methods: A cross-sectional study comprises 17 CF patients and 18 healthy controls. Adiponectin levels were measured by magnetic bead-based multiplex assay. Results: The mean age of adult CF patients was 22.9 years± 3.8 (18-30) and 76.5% CF patients had pancreatic sufficiency. The mean BMI in healthy controls was slightly higher than CF patients. The mean sputum adiponectin level was significantly lower than plasma adiponectin levels in CF patients and healthy controls (p<0.001), whereas no significant difference in plasma adiponectin levels between CF patients and healthy controls. The mean sputum adiponectin level was observed to be higher in CF patients with pancreatic insufficiency. Sputum adiponectin level was correlated positively with plasma adiponectin level in CF patients (r= 0.47, p=0.06). Sputum and plasma adiponectin levels in CF patients were correlated negatively with BMI and percentage predicted forced expiratory volume in 1 second (FEV1) and Forced vital capacity (FVC). Conclusions: Sputum adiponectin may provide a minimally invasive tool in the assessment of an inflammatory status in CF patients. Further larger study to address any difference in sputum and plasma adiponectin levels among CF patients with pancreatic sufficiency versus pancreatic insufficiency.

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Cost-effectiveness of cell salvage and donor blood transfusion during caesarean section: results from a randomised controlled trial

BMJ Open ◽

10.1136/bmjopen-2018-022352 ◽

2019 ◽

Vol 9 (2) ◽

pp. e022352 ◽

Cited By ~ 4

Author(s):

Carol McLoughlin ◽

Tracy E Roberts ◽

Louise J Jackson ◽

Philip Moore ◽

Matthew Wilson ◽

...

Keyword(s):

Cost Effectiveness ◽

Caesarean Section ◽

Blood Transfusion ◽

Controlled Trial ◽

Mean Difference ◽

Cell Salvage ◽

Donor Blood ◽

Intention To Treat ◽

The Mean ◽

Randomised Controlled

ObjectivesTo evaluate the cost-effectiveness of routine use of cell salvage during caesarean section in mothers at risk of haemorrhage compared with current standard of care.DesignModel-based cost-effectiveness evaluation alongside a multicentre randomised controlled trial. Three main analyses were carried out on the trial data: (1) based on the intention-to-treat principle; (2) based on the per-protocol principle; (3) only participants who underwent an emergency caesarean section.Setting26 obstetric units in the UK.Participants3028 women at risk of haemorrhage recruited between June 2013 and April 2016.InterventionsCell salvage (intervention) versus routine care without salvage (control).Primary outcome measuresCost-effectiveness based on incremental cost per donor blood transfusion avoided.ResultsIn the intention-to-treat analysis, the mean difference in total costs between cell salvage and standard care was £83. The estimated incremental cost-effectiveness ratio (ICER) was £8110 per donor blood transfusion avoided. For the per-protocol analysis, the mean difference in total costs was £92 and the ICER was £8252. In the emergency caesarean section analysis, the mean difference in total costs was £55 and the ICER was £13 713 per donor blood transfusion avoided. This ICER is driven by the increased probability that these patients would require a higher level of postoperative care and additional surgeries. The results of these analyses were shown to be robust for the majority of deterministic sensitivity analyses.ConclusionsThe results of the economic evaluation suggest that while routine cell salvage is a marginally more effective strategy than standard care in avoiding a donor blood transfusion, there is uncertainty in relation to whether it is a less or more costly strategy. The lack of long-term data on the health and quality of life of patients in both arms of the trial means that further research is needed to fully understand the cost implications of both strategies.Trial registration numberISRCTN66118656.

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Nocturnal hyperalimentation via a gastrostomy tube in children with cystic fibrosis

Voprosy praktičeskoj pediatrii ◽

10.20953/1817-7646-2021-2-22-34 ◽

2021 ◽

Vol 16 (2) ◽

pp. 22-34

Author(s):

I. Sokolov ◽

◽

E.A. Roslavtseva ◽

V.P. Chernevich ◽

N.I. Burkina ◽

...

Keyword(s):

Cystic Fibrosis ◽

Lung Function ◽

Nutritional Status ◽

Body Weight Gain ◽

Forced Expiratory Volume ◽

Gastrostomy Tube ◽

Percutaneous Endoscopic Gastrostomy Tube ◽

Endoscopic Control ◽

Low Profile ◽

The Mean

Objective. To assess the efficacy of nocturnal hyperalimentation via a low-profile gastrostomy tube for in the improvement of nutritional status and lung function in children with severe cystic fibrosis (CF). Patients and methods. We used nocturnal hyperalimentation with a nutrient solution via a low-profile gastrostomy tube installed under endoscopic control to improve nutritional status of 16 CF children with severe protein-calorie malnutrition aged between 5.5 and 17.5 years. The mean duration of nocturnal hyperalimentation was 1.3 years (range: 7 months–54 months). The nutritional status was evaluated using the WHOAntho Plus software; pulmonary function was evaluated by assessing forced expiratory volume in 1 minute (FEV1) and the number of bronchopulmonary exacerbations per year. Results. All children demonstrated an improvement in their weight and height. After the first 12 months, the mean body weight gain was 5 kg; during the second and third years, children gained 4 kg and nearly 2 kg, respectively. The mean increase in height was 7 cm in the first year, 10 cm in the second year, and 4 cm in the third year. All patients except one had an increase in FEV1 and fewer exacerbations. Complications after gastrostomy were rare and easily addressed. Conclusion. Nocturnal hyperalimentation via a percutaneous endoscopic gastrostomy tube is an effective and safe method of improving nutritional status in children with CF, which delays the progression of bronchopulmonary lesions and lung function reduction, improves the prognosis and quality of life. Key words: cystic fibrosis, children, nutritional status, lung function, enteral nutrition, low-profile gastrostomy tube, nocturnal hyperalimentation

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Continuous versus Intermittent Infusions of Ceftazidime for Treating Exacerbation of Cystic Fibrosis

Antimicrobial Agents and Chemotherapy ◽

10.1128/aac.00174-09 ◽

2009 ◽

Vol 53 (9) ◽

pp. 3650-3656 ◽

Cited By ~ 44

Author(s):

Dominique Hubert ◽

Evelyne Le Roux ◽

Thibaud Lavrut ◽

Benoit Wallaert ◽

Philippe Scheid ◽

...

Keyword(s):

Cystic Fibrosis ◽

Continuous Infusion ◽

Antibiotic Treatment ◽

Forced Expiratory Volume ◽

Trough Concentrations ◽

The Mean ◽

Treatment Procedures ◽

Susceptibility Profiles ◽

Infusion Regimen

ABSTRACT The present multicenter, randomized crossover study compared the safety and efficacy of continuous infusion with those of short infusions of ceftazidime in patients with cystic fibrosis. Patients with chronic Pseudomonas aeruginosa colonization received two successive courses of intravenous tobramycin and ceftazidime (200 mg/kg of body weight/day) for pulmonary exacerbation administered as thrice-daily short infusions or as a continuous infusion. The primary endpoint was the variation in the forced expiratory volume in 1 s (FEV1) during the course of antibiotic treatment. Sixty-nine of the 70 patients enrolled in the study received at least one course of antibiotic treatment. The improvement in FEV1 at the end of therapy was not statistically different between the two treatment procedures (+7.6% after continuous infusion and +5.5% after short infusions) but was better after continuous ceftazidime treatment in patients harboring resistant isolates (P < 0.05). The interval between the course of antibiotic treatments was longer after the continuous infusion than after the short infusion of ceftazidime (P = 0.04). The mean serum ceftazidime concentration during the continuous infusion was 56.2 ± 23.2 μg/ml; the mean peak and trough concentrations during the short infusions were 216.3 ± 71.5 and 12.1 ± 8.7 μg/ml, respectively. The susceptibility profiles of the P. aeruginosa isolates remained unchanged and were similar for both regimens. Quality-of-life scores were similar whatever the treatment procedure, but 82% of the patients preferred the continuous-infusion regimen. Adverse events were not significantly different between the two regimens. In conclusion, the continuous infusion of ceftazidime did not increase its toxicity and appeared to be as efficient as short infusions in patients with cystic fibrosis as a whole, but it gave better results in patients harboring resistant isolates of P. aeruginosa.

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Unique Pattern of Pulmonary Function after Exercise in Patients with Cystic Fibrosis

Pediatric Exercise Science ◽

10.1123/pes.6.3.275 ◽

1994 ◽

Vol 6 (3) ◽

pp. 275-286 ◽

Cited By ~ 1

Author(s):

Ted. A. Kaplan ◽

Gina Moccia ◽

Robert M. McKey

Keyword(s):

Cystic Fibrosis ◽

Pulmonary Function ◽

Time Course ◽

Forced Expiratory Volume ◽

Distinct Pattern ◽

Degree Pattern ◽

Function Test ◽

The Mean ◽

Exercise Induced ◽

Mean Time

The purpose of this study was to assess the incidence, degree, pattern, and time course of pulmonary function test (PFT) changes measured after a dynamic exercise challenge. Forced expiratory maneuvers were performed before and serially after a 7-min run-walk in 44 patients with cystic fibrosis (CF). Twenty-four patients met at least one PFT criterion for exercise-induced bronchospasm (EIB) within 21 min after exercise, and 38 patients had >5% increase in a PFT parameter at some point after exercise. The mean time of greatest increase in PFTs occurred sooner than the greatest decrease, which was later than for 22 patients with asthma or allergic rhinitis found to have EIB. Age was inversely related to percentage fall in forced expiratory volume in 1 s (FEV,) after exercise. These results demonstrate a distinct pattern of PFTs measured after exercise in most patients with CF, with an early bronchodilation followed by a significant decrease in PFTs.

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