scholarly journals Eliciting health state utilities for Aromatic L-amino Acid Decarboxylase (AADC) deficiency: a UK vignette study

2021 ◽  
Vol 5 (1) ◽  
Author(s):  
Adam B. Smith ◽  
Andria Hanbury ◽  
Katharina Buesch
Keyword(s):  
Amino Acid ◽  
Task Completion ◽  
Acid Decarboxylase ◽  
Health State ◽  
Health States ◽  
Amino Acid Decarboxylase ◽  
Vignette Study ◽  
Young Infants ◽  
Health State Utilities ◽  
Head Control

Abstract Purpose The aim of this study was to generate health state utilities for aromatic L-amino acid decarboxylase (AADC) deficiency, a rare genetic, lifelong neurogenerative condition predominantly manifesting in young infants. Methods Participants were presented with health state vignettes. These had been previously developed based on published literature, clinician input, interviews with parents of AADC deficiency patients and expert opinion. A total of 5 health state vignettes were presented: bedridden, head control, sitting unsupported, standing with assistance and walking with assistance. Health state utilities (HSU) were elicited using time-trade off (TTO; 10-year time horizon) and the standard gamble (SG). The vignettes were completed online by panel participants drawn from a representative sample of the United Kingdom residential population. Results A total of 1598 participants completed the vignettes. Around 21% had incongruent responses (higher utilities for the bedridden compared to walking health states). Incongruent responses were associated with shorter task completion times, gender and parental status. These responses were removed from the analysis. Health state utilities (HSU) increased correspondingly as health states improved for both the TTO and SG. The mean HSU (standard deviation) for the TTO task were: bedridden state 0.49 (0.34); head control 0.54 (0.33), sitting unsupported 0.63 (0.31); standing with assistance 0.68 (0.31); and walking with assistance 0.73 (0.31). For the SG, mean health state utilities were: 0.56 (0.28), 0.57 (0.27), 0.67 (0.24), 0.70 (0.24), and 0.75 (0.25), respectively. Conclusion Health state utilities were derived for AADC deficiency through a vignette study. These will be used for a cost-effectiveness model of an AADC deficiency treatment.

scholarly journals Eliciting Health State Utilities for Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency: A Vignette Study in France

2021 ◽  
Vol Volume 12 ◽  
pp. 237-246
Author(s):  
Adam B Smith ◽  
Andria Hanbury ◽  
Igor Beitia Ortiz de Zarate ◽  
Florence Hammes ◽  
Gerard de Pouvourville ◽  
...  
Keyword(s):  
Amino Acid ◽  
Acid Decarboxylase ◽  
Health State ◽  
Amino Acid Decarboxylase ◽  
Vignette Study ◽  
Health State Utilities

scholarly journals Aromatic L-amino Acid Decarboxylase (AADC) deficiency: results from an Italian modified Delphi consensus

2021 ◽  
Vol 47 (1) ◽  
Author(s):  
Carlo Fusco ◽  
◽  
Vincenzo Leuzzi ◽  
Pasquale Striano ◽  
Roberta Battini ◽  
...  
Keyword(s):  
Amino Acid ◽  
Diagnostic Delay ◽  
Steering Committee ◽  
Outcome Data ◽  
Diagnosis And Treatment ◽  
Acid Decarboxylase ◽  
Delphi Consensus ◽  
Amino Acid Decarboxylase ◽  
Modified Delphi ◽  
Number Of Patients

Abstract Background Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare and underdiagnosed neurometabolic disorder resulting in a complex neurological and non-neurological phenotype, posing diagnostic challenges resulting in diagnostic delay. Due to the low number of patients, gathering high-quality scientific evidence on diagnosis and treatment is difficult. Additionally, based on the estimated prevalence, the number of undiagnosed patients is likely to be high. Methods Italian experts in AADC deficiency formed a steering committee to engage clinicians in a modified Delphi consensus to promote discussion, and support research, dissemination and awareness on this disorder. Five experts in the field elaborated six main topics, each subdivided into 4 statements and invited 13 clinicians to give their anonymous feedback. Results 100% of the statements were answered and a consensus was reached at the first round. This enabled the steering committee to acknowledge high rates of agreement between experts on clinical presentation, phenotypes, diagnostic work-up and treatment strategies. A research gap was identified in the lack of standardized cognitive and motor outcome data. The need for setting up an Italian working group and a patients’ association, together with the dissemination of knowledge inside and outside scientific societies in multiple medical disciplines were recognized as critical lines of intervention. Conclusions The panel expressed consensus with high rates of agreement on a series of statements paving the way to disseminate clear messages concerning disease presentation, diagnosis and treatment and strategic interventions to disseminate knowledge at different levels. Future lines of research were also identified.

Changes in serotonin contents in brain affect metabolic heat production of rabbits in cold

1978 ◽  
Vol 235 (1) ◽  
pp. R41-R47
Author(s):  
M. T. Lin ◽  
I. H. Pang ◽  
S. I. Chern ◽  
W. Y. Chia
Keyword(s):  
Blood Flow ◽  
Amino Acid ◽  
Heat Loss ◽  
Acid Decarboxylase ◽  
Evaporative Heat Loss ◽  
Decarboxylase Inhibitor ◽  
Ambient Temperatures ◽  
Amino Acid Decarboxylase ◽  
Metabolic Heat ◽  
Normal Limits

Elevating serotonin (5-HT) contents in brain with 5-hydroxytryptophan (5-HTP) reduced rectal temperature (Tre) in rabbits after peripheral decarboxylase inhibition with the aromatic-L-amino-acid decarboxylase inhibitor R04-4602 at two ambient temperatures (Ta), 2 and 22 degrees C. The hypothermia was brought about by both an increase in respiratory evaporative heat loss (Eres) and a decrease in metabolic rate (MR) in the cold. At a Ta of 22 degrees C, the hypothermia was achieved solely due to an increase in heat loss. Depleting brain contents of 5-HT with intraventricular, 5,7-dihydroxytryptamine (5,7-DHT) produced an increased Eres and ear blood flow even at Ta of 2 degrees C. Also, MR increased at all but the Ta of 32 degrees C. However, depleting the central and peripheral contents of 5-HT with p-chlorophenylalanine (pCPA) produced lower MR accompanied by lower Eres in the cold compared to the untreated control. Both groups of pCPA-treated and 5,7-DHT-treated animals maintained their Tre within normal limits. The data suggest that changes in 5-HT content in brain affects the MR of rabbits in the cold. Elevating brain content of 5-HT tends to depress the MR response to cold, while depleting brain content of 5-HT tends to enhance the MR response to cold.

scholarly journals NCOG-36. FIRST HEALTH UTILITIES OF GLIOBLASTOMA PATIENTS USING TTFIELDS TREATMENT

2020 ◽  
Vol 22 (Supplement_2) ◽  
pp. ii137-ii137
Author(s):  
Gordon Chavez ◽  
Christina Proescholdt
Keyword(s):  
Population Sample ◽  
National Health System ◽  
Medical Decision ◽  
Economic Evaluations ◽  
Health State ◽  
High Quality ◽  
Health States ◽  
Tumor Treating Fields ◽  
Health State Utilities ◽  
The Uk

Abstract BACKGROUND Despite the importance of Health State Utilities for health policy and medical decision making, there are no publications that provide high quality utility values estimated from glioblastoma multiforme (GBM) patients. Published health economic evaluations for GBM treatments rely on utilities determined by Garside et al. (2007), which used the standard gamble method in healthy panel members of the UK National Health System. There are no published utilities for GBM estimated from a general population sample, and there are no utility estimates whatsoever for Tumor Treating Fields (TTFields) users. METHODS We designed a study to remedy this major deficit by eliciting utilities directly from GBM patients using the EuroQol 5-Dimension (EQ-5D) survey. The EQ-5D is a widely used and NICE-recommended tool for the estimation of health state utilities. The survey is composed of a questionnaire that asks patients to specify their health state along 5 dimensions: Mobility, Self-Care, Usual Activities, Pain/Discomfort, and Anxiety/Depression. Statistical models provided by EuroQol’s network of researchers convert this data into health state utility estimates. RESULTS The EQ-5D questionnaire is administered to active patients using TTFields treatment during the study duration, allowing the elicitation of health preference measures for different glioblastoma health states based on: progression status (progressed vs. non-progressed), current treatments (TTFields only vs. TTFields + others) and time-from-diagnosis (0-12 months vs. > 12 months) CONCLUSION These results are important for understanding the patient preferences using TTFields treatment and communicating these preferences to decision makers. This study is the first to provide direct, high quality utility measures in glioblastoma patients using TTFields treatment.

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