Patient involvement to inform the design of a clinical trial in postbariatric hypoglycaemia

Abstract Background Bariatric surgery may lead to symptomatic postprandial hypoglycaemia as a major side effect without established therapy so far. We aimed to develop an evidence-based study design of a clinical trial that tests treatment options and can provide useful patient-relevant evidence. Methods We searched systematically for guidance of core outcome sets to determine the most relevant types of outcomes and duration of such a trial. Our search comprised literature databases, a database of core outcome sets and self-help organizations. We then developed a simple online questionnaire based on interviews with German-speaking patients with postprandial hypoglycaemia after bariatric surgery. We recruited participants by reaching out to all German speaking endocrinologists in Switzerland and large Swiss bariatric centres. We asked for preferences regarding outcome types and acceptable duration of being included in a corresponding clinical trial. Results The literature search did not identify evidence-based guidance for informing our study design. Experience of clinical and research routine as well as patient interviews helped in identifying potential outcomes and the design of an online questionnaire. Therein, a total of 29 persons started the questionnaire and 22 answered questions related to the primary outcome. Of these, 17 (77.3%) deemed quality of life more relevant as primary outcome than the rate of hypoglycaemic episodes. A trial length of four weeks or longer was regarded as acceptable for 19 of 21 respondents to this question (91.4%) and of six months or longer for 12 respondents (56%). Conclusions In situations with no other guidance, a simple questionnaire may help to inform trial design decisions. This study identifies a patient preference for “quality of life” as a primary outcome and supports the evidence-based conception of a patient-centred clinical trial in postbariatric hypoglycaemia.

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Patient involvement to inform the design of a clinical trial in postbariatric hypoglycaemia

10.21203/rs.3.rs-55915/v2 ◽

2021 ◽

Author(s):

Matthias Hepprich ◽

Marc Y Donath ◽

Lars G Hemkens

Keyword(s):

Quality Of Life ◽

Bariatric Surgery ◽

Clinical Trial ◽

Primary Outcome ◽

Evidence Based ◽

Core Outcome ◽

Online Questionnaire ◽

Core Outcome Sets ◽

German Speaking

Abstract Background: Bariatric surgery may lead to symptomatic postprandial hypoglycaemia as a major side effect without established therapy so far. We aimed to develop an evidence-based study design of a clinical trial that tests treatment options and can provide useful patient-relevant evidence.Methods: We searched systematically for guidance of core outcome sets to determine the most relevant types of outcomes and duration of such a trial. Our search comprised literature databases, a database of core outcome sets and self-help organizations. We then developed a simple online questionnaire based on interviews with German-speaking patients with postprandial hypoglycaemia after bariatric surgery. We recruited participants by reaching out to all German speaking endocrinologists in Switzerland and large Swiss bariatric centres. We asked for preferences regarding outcome types and acceptable duration of being included in a corresponding clinical trial.Results: The literature search did not identify evidence-based guidance for informing our study design. Experience of clinical and research routine as well as patient interviews helped in identifying potential outcomes and the design of an online questionnaire. Therein, a total of 29 persons started the questionnaire and 22 answered questions related to the primary outcome. Of these, 17 (77.3%) deemed quality of life more relevant as primary outcome than the rate of hypoglycaemic episodes. A trial length of four weeks or longer was regarded as acceptable for 19 of 21 respondents to this question (91.4 %) and of six months or longer for 12 respondents (56 %). Conclusions: In situations with no other guidance, a simple questionnaire may help to inform trial design decisions. This study identifies a patient preference for “quality of life” as a primary outcome and supports the evidence-based conception of a patient-centred clinical trial in postbariatric hypoglycaemia.

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Patient Involvement to Inform the Design of a Clinical Trial in Postbariatric Hypoglycemia

10.21203/rs.3.rs-55915/v1 ◽

2020 ◽

Author(s):

Matthias Hepprich ◽

Marc Y Donath ◽

Lars G Hemkens

Keyword(s):

Quality Of Life ◽

Bariatric Surgery ◽

Clinical Trial ◽

Primary Outcome ◽

Evidence Based ◽

Core Outcome ◽

Online Questionnaire ◽

Core Outcome Sets ◽

German Speaking

Abstract Background: Bariatric surgery may lead to symptomatic postprandial hypoglycemia as a major side effect without established therapy so far. We aimed to develop an evidence-based study design of a clinical trial that tests treatment options and can provide useful patient-relevant evidence.Methods: We searched systematically for guidance of core outcome sets to determine the most relevant types of outcomes and duration of such a trial. Our search comprised literature databases, a database of core outcome sets and self-help organizations. We then developed a simple online questionnaire based on interviews with German-speaking patients with postprandial hypoglycemia after bariatric surgery. We recruited participants by reaching out to all German speaking endocrinologists in Switzerland and large Swiss bariatric centers. We asked for preferences regarding outcome types and acceptable duration of being included in a corresponding clinical trial.Results: The systematic search did not identify evidence-based guidance for informing our study design. Experience of clinical and research routine as well as patient interviews helped in identifying potential outcomes and the design of an online questionnaire. Therein, a total of 29 persons started the questionnaire and 22 answered questions related to the primary outcome. Of these, 17 (77.3%) deemed quality of life more relevant as primary outcome than the rate of hypoglycemic episodes. A trial length of four weeks or longer was regarded as acceptable for 19 of 21 respondents to this question (91.4 %) and of six months or longer for 12 respondents (56 %). Conclusions: In situations with no other guidance, a simple questionnaire may help to inform trial design decisions. This study identifies a patient preference for “quality of life” as a primary outcome and supports the evidence-based conception of a patient-centered clinical trial in postbariatric hypoglycemia.

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Medically tailored meals for the management of symptomatic ascites: the SALTYFOOD pilot randomized clinical trial

Gastroenterology Report ◽

10.1093/gastro/goaa059 ◽

2020 ◽

Author(s):

Elliot B Tapper ◽

Jad Baki ◽

Samantha Nikirk ◽

Scott Hummel ◽

Sumeet K Asrani ◽

...

Keyword(s):

Quality Of Life ◽

Clinical Trial ◽

Clinical Management ◽

Primary Outcome ◽

Standard Of Care ◽

Protein Supplement ◽

Sodium Diet ◽

Low Sodium Diet ◽

Low Sodium

Abstract Background Ascites is a costly, morbid complication of cirrhosis. Although a low-sodium diet is central to the clinical management of ascites, its efficacy is limited by poor adherence. We aimed to determine the feasibility and impact of low-sodium medically tailored meals (MTM) intervention. Methods We enrolled 40 persons with cirrhosis and ascites at the time of a paracentesis in a 12-week, 1:1 randomized trial of standard of care (SOC) (low-sodium diet educational handout) or MTM with <2,000 mg of sodium, >2,100 kcal, and >80 g of protein including a nocturnal protein supplement. We determined the proportion of eligible candidates recruited and adherence to MTM. The primary outcome was the number of paracenteses performed during weeks 0–12. We also collected ascites-specific quality-of-life (ASI-7) scores. Results The median age of the enrolled subjects was 54 (IQR, 47–63) years, 46% were female, with median MELD-Na 18 (IQR, 11–23) and albumin 2.7 (IQR, 2.5–3.3) g/dL. At baseline, subjects reported a median of two (IQR, 1–3) paracenteses in the prior 4 weeks. Adherence to the meal schedule was excellent save for when hospitalizations occurred. After 12 weeks, patients in the MTM arm required fewer paracenteses per week than those in the SOC group [median (IQR): 0.34 (0.14–0.54) vs 0.45 (0.25–0.64)]. During the trial, four (20%) SOC patients died, whereas two (10%) died and one (5%) was transplanted in the MTM arm. Ascites-specific quality of life improved to a greater degree in the MTM arm compared to the SOC arm, by 25% (IQR, –11% to 61%) vs 13% (IQR, –28% to 54%). Conclusion A trial of MTM for persons with ascites is feasible and potentially effective. Both arms experienced benefits, highlighting the role for improved education and closer monitoring in this challenging condition.

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Entwicklung und Anwendung der Lebensqualitätserfassung in der Dermatologie

Karger Kompass Dermatologie ◽

10.1159/000505472 ◽

2020 ◽

Vol 8 (1) ◽

pp. 6-10 ◽

Cited By ~ 1

Author(s):

Pavel V. Chernyshov

Keyword(s):

Quality Of Life ◽

Klinische Studien ◽

Dermatology Life Quality Index ◽

Quality Index ◽

Life Quality ◽

Core Outcome ◽

Core Outcome Sets ◽

Life Quality Index

Die Entwicklung des Dermatology Life Quality Index (DLQI) Fragebogens erleichterte die Durchführung zahlreicher Studien zu den Auswirkungen von Hautkrankheiten auf die Lebensqualität (quality of life, QoL) der Patienten. Viele nationale und internationale Leitlinien empfehlen die Erfassung der Lebensqualität in der Dermatologie und einige enthalten detaillierte Empfehlungen zu den Behandlungszielen und Änderungen von Behandlungsansätzen entsprechend den DLQI-Score-Gruppen und dem geringsten klinisch relevanten Unterschied. Die Methoden zur Entwicklung und Validierung von QoL-Instrumenten werden kontinuierlich strenger. Initiativen zur Auswahl von Core Outcome Sets für Hautkrankheiten sind auf klinische Studien ausgerichtet, können aber auch für Kliniker hilfreich sein. Die Verwendung von QoL-Daten in der klinischen Praxis ist mit verschiedenen Vorteilen verbunden, doch liegen derzeit nur sehr begrenzte Erfahrungen damit vor. Die Erfassung der Lebensqualität in der Dermatologie entwickelt sich rasant und verlagert sich allmählich von der Theorie in die Praxis.

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A single-center randomized clinical trial of palliative rehabilitation versus standard care alone in patients with newly diagnosed non-resectable cancer.

Journal of Clinical Oncology ◽

10.1200/jco.2018.36.34_suppl.75 ◽

2018 ◽

Vol 36 (34_suppl) ◽

pp. 75-75 ◽

Cited By ~ 1

Author(s):

Lise Nottelmann ◽

Mogens Groenvold ◽

Morten Aagard Petersen ◽

Tove Vejlgaard ◽

Lars Henrik Jensen

Keyword(s):

Quality Of Life ◽

Clinical Trial ◽

Palliative Care ◽

Standard Care ◽

Primary Outcome ◽

Absolute Difference ◽

Group Program ◽

Rehabilitation Intervention ◽

Oncology Care

75 Background: Palliative care and rehabilitation may both improve quality of life, but research on their combination and early integration into oncology care is sparse. Methods: Adults diagnosed with non-resectable cancers within the last 8 weeks were randomized to standard oncology care alone or an additional offer of individually tailored palliative rehabilitation. Two mandatory consultations and a 12 week open contact with a specialized palliative care team were offered. An additional opportunity was participation in a multidisciplinary group program combining a patient/caregiver school with physical exercise, individual consultations, or both. Participants were assessed at baseline and after 6 and 12 weeks with an extended version of the EORTC QLQ-C30 questionnaire using the item banks for computer-adaptive testing to obtain improved measurement. At baseline participants were asked to choose what they needed help with the most from a list of possible 'primary problems' corresponding to 12 of the 15 QLQ-C30 scales. The primary outcome was the change in that "primary problem" scale measured as area under the curve across the 12 weeks. Group differences were tested in an adjusted linear regression model. Results: 301 patients with lung- (40%), gastrointestinal- (27%), prostatic- (18%), and various other solid tumors (15%) were included. 139 patients were allocated to the intervention group and 149 to the standard care group. The palliative rehabilitation intervention was received by 132. Of those, 26 received the two mandatory consultations only, 59 additionally participated in a group program, and 47 additionally received individual consultations without participation in a group. The intervention showed an effect for the primary outcome with an absolute between-group difference of 3.0 (0.0;6.0) p = 0.047. The result was confirmed by a sensitivity analysis of the change from baseline to 12 weeks showing an absolute difference of 3.3 (1.0; 5.6) p = 0.005. Conclusions: A palliative rehabilitation intervention initiated soon after diagnosis and integrated in the standard oncology treatment improved quality of life. Clinical trial information: NCT02332317.

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Misophonia: An Evidence-Based Case Report

American Journal of Audiology ◽

10.1044/2020_aja-19-00111 ◽

2020 ◽

Vol 29 (4) ◽

pp. 685-690

Author(s):

C. S. Vanaja ◽

Miriam Soni Abigail

Keyword(s):

Quality Of Life ◽

Case Report ◽

Case History ◽

Pure Tone ◽

Physiological Responses ◽

Evidence Based ◽

Management Option ◽

Pure Tone Audiogram

Purpose Misophonia is a sound tolerance disorder condition in certain sounds that trigger intense emotional or physiological responses. While some persons may experience misophonia, a few patients suffer from misophonia. However, there is a dearth of literature on audiological assessment and management of persons with misophonia. The purpose of this report is to discuss the assessment of misophonia and highlight the management option that helped a patient with misophonia. Method A case study of a 26-year-old woman with the complaint of decreased tolerance to specific sounds affecting quality of life is reported. Audiological assessment differentiated misophonia from hyperacusis. Management included retraining counseling as well as desensitization and habituation therapy based on the principles described by P. J. Jastreboff and Jastreboff (2014). A misophonia questionnaire was administered at regular intervals to monitor the effectiveness of therapy. Results A detailed case history and audiological evaluations including pure-tone audiogram and Johnson Hyperacusis Index revealed the presence of misophonia. The patient benefitted from intervention, and the scores of the misophonia questionnaire indicated a decrease in the severity of the problem. Conclusions It is important to differentially diagnose misophonia and hyperacusis in persons with sound tolerance disorders. Retraining counseling as well as desensitization and habituation therapy can help patients who suffer from misophonia.

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Rationale, baseline characteristics and methodology of the non-interventional VIVA study in postmenopausal osteoporosis

Osteologie/Osteology ◽

10.1055/s-0037-1620027 ◽

2014 ◽

Vol 23 (01) ◽

pp. 49-55

Author(s):

L. C. Hofbauer ◽

D. Felsenberg ◽

M. Amling ◽

A. Kurth ◽

P. Hadji

Keyword(s):

Quality Of Life ◽

Postmenopausal Osteoporosis ◽

Primary Outcome ◽

Osteoporosis Treatment ◽

Secondary Outcome ◽

Matched Pairs ◽

Fracture History ◽

Baseline Characteristics ◽

Study Inclusion

SummaryIt is important to understand compliance and persistence with medication use in the clinical practice of osteoporosis treatment. The purpose of this work is to describe the “intravenous ibandronate versus oral alendronate” (VIVA) study, a non-interventional trial to assess the compliance and persistence of osteopenic postmenopausal women with treatment via weekly oral alendronate or intravenous ibandronate (Bonviva®) every three months.4477 patients receiving ibandronate 3 mg i. v. quarterly and 1491 patients receiving alendronate 70 mg orally weekly were included in the study. Matched pairs of 901 subjects in each group were also generated. Matching was performed on the basis of age, body mass index, fracture history at study inclusion, prior treatment with bisphosphonates and the number of concomitant disorders. Secondary outcome measures of osteoporosis related fractures, mobility restriction and pain, analgesia, quality of life questionnaires as well as attitudes to medications were assessed. The primary outcome parameters of compliance and persistence will be tracked in these subjects.At baseline, the entire collectives differed significantly on body weight (less in ibandronate group), duration since osteo - porosis diagnosis (longer in ibandronate), and incidence of prior osteoporotic fracture (higher in ibandronate group). The matched-pairs differed only on mobility restriction and quality of life (both worse in ibandronate group).The results from the VIVA study trial will provide scientific rationale for clinical recommendations in the pharmacological treatment of postmenopausal osteoporosis.

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