The Experts below are selected from a list of 87 Experts worldwide ranked by ideXlab platform
J Giesinger - One of the best experts on this subject based on the ideXlab platform.
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International validation of the EORTC QLQ-ELD14 questionnaire for assessment of health-related quality of life elderly patients with cancer
British Journal of Cancer, 2013Co-Authors: S Wheelwright, A-s Darlington, D Fitzsimmons, P Fayers, J I Arraras, F Bonnetain, E Brain, A Bredart, W-c Chie, J GiesingerAbstract:Background: Older people represent the majority of cancer patients but their specific needs are often ignored in the development of health-related quality of life (HRQOL) instruments. The European Organisation for Research and Treatment of Cancer (EORTC) QLQ-ELD15 was developed to supplement the EORTC’s core questionnaire, the QLQ-C30, for measuring HRQOL in patients aged >70 years in oncology studies. Methods: Patients ( n =518) from 10 countries completed the QLQ-C30, QLQ-ELD15 and a Debriefing Interview. Eighty two clinically stable patients repeated the questionnaires 1 week later (test–retest analysis) and 107 others, with an expected change in clinical status, repeated the questionnaires 3 months later (response to change analysis, RCA). Results: Information from the Debriefing Interview, factor analysis and item response theory analysis resulted in the removal of one item (QLQ-ELD15→QLQ-ELD14) and revision of the proposed scale structure to five scales (mobility, worries about others, future worries, maintaining purpose and illness burden) and two single items (joint stiffness and family support). Convergent validity was good. In known-group comparisons, the QLQ-ELD14 differentiated between patients with different disease stage, treatment intention, number of comorbidities, performance status and geriatric screening scores. Test–retest and RCA analyses were equivocal. Conclusion: The QLQ-ELD14 is a validated HRQOL questionnaire for cancer patients aged ⩾70 years. Changes in elderly patients’ self-reported HRQOL may be related to both cancer evolution and non-clinical events.
Benjamin L. Shneider - One of the best experts on this subject based on the ideXlab platform.
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Development of a Novel Tool to Assess the Impact of Itching in Pediatric Cholestasis
The Patient - Patient-Centered Outcomes Research, 2018Co-Authors: Binita M. Kamath, Linda Abetz-webb, Ciara Kennedy, Bonnie Hepburn, Martha Gauthier, Nathan Johnson, Sharon Medendorp, Alejandro Dorenbaum, Lora Todorova, Benjamin L. ShneiderAbstract:Objectives The aim was to develop a clinical outcome assessment (COA) for itching in children with cholestatic pruritus. Methods This prospective study aimed to enroll patients aged 4–30 years with Alagille syndrome (ALGS) or progressive familial intrahepatic cholestasis type 1 and caregivers of patients aged 5 months to 14 years. Eligible patients experienced itching during ≥3 of the 7 days before enrollment and had not undergone liver transplant or surgical interruption of the enterohepatic circulation. Open-ended qualitative Interviews confirmed that itching was a primary concern for patients and caregivers. Diaries were modified and then evaluated by participants during cognitive Debriefing. Interview results were reviewed by clinical, COA and statistical experts. Diary questions were revised following an interim analysis before finalizing the Itch Reported Outcome (ItchRO). Results Thirty-six Interviews were analyzed, representing 25 families of patients with ALGS. Itching was reported spontaneously (without prompting by the Interviewer) by ten of 12 patients with ALGS and 19 of 20 caregivers. Consequences of itching included skin damage (78%), mood changes (59%), and difficulties staying asleep (59%) or falling asleep (53%). Two versions of the ItchRO were developed: ItchRO(Patient) for self-completion by patients and ItchRO(Observer) for caregivers. The ItchRO diaries comprise a single scorable item to assess itch and are to be completed twice daily (morning and evening). Conclusions Itching was the most bothersome ALGS symptom reported by study participants. We have developed the ItchRO(Patient) and ItchRO(Observer) to assess itching in children with ALGS and other cholestatic liver diseases. These diaries are being validated for use in clinical trials.
Jens Panse - One of the best experts on this subject based on the ideXlab platform.
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Design and development of a disease-specific quality of life tool for patients with aplastic anaemia and/or paroxysmal nocturnal haemoglobinuria (QLQ-AA/PNH)—a report on phase III
Annals of Hematology, 2019Co-Authors: Cathrin Niedeggen, Martha Groth, Susanne Singer, Andrea Petermann-meyer, Britta Höchsmann, Hubert Schrezenmeier, Tim H Brummendorf, Alexander Roth, Jens PanseAbstract:To date, instruments to measure quality of life (QoL) specifically for patients with acquired aplastic anaemia (AA) and paroxysmal nocturnal haemoglobinuria (PNH) are lacking altogether. As a consequence, this issue is either underevaluated or alternatively, instruments originally designed for cancer patients are being used. We therefore started to systematically develop a AA/PNH-specific QoL (QLQ-AA/PNH) instrument in these ultra-rare diseases according to European Organisation for Research and Treatment of Cancer (EORTC) guidelines. While phases I and II of the process have previously been published, we now report on the resulting instrument (phase III of this process). As part of the phase III of the evaluation process, we approached patients through physicians, patient support groups, and patient conferences. After participants completed the preliminary questionnaire and reported socio-demographic data, they were Interviewed in person or via phone with a Debriefing Interview to find out whether the items were relevant, easy to understand, and acceptable to patients and whether there was anything missing in the questionnaire. We hypothesised what items could be combined into a scale and calculated Cronbach’s alpha to define its preliminary internal consistency. After definition of a priori criteria to keep or delete items, a group of six experts met in person, discussed the results, and decided on in- or exclusion. A total of 48 patients were enrolled, 21 of those suffered from AA (44%), 13 from PNH (27%), and 14 from AA/PNH syndrome (29%). The median time to complete the 69 items was 10 min (range 5–20), mean time 11 min. The compliance criterion (> 95% completion) was fulfilled by 57 items. Twenty-three items were mentioned as especially relevant by ≥ 2% of the patients. Cronbach’s alpha of the hypothesised scales ranged from 0.63 (social support) to 0.92 (fear of progression and illness intrusiveness). Finally, 47 items were kept; 16 were deleted, and 5 were changed, while 1 item expanded. This resulted in 54 items in total. As no issues were mentioned to lacking by a minimum of five patients, no items were added to the questionnaire. After completion, the AA/PNH-QoL tool (QLQ-AA/PNH) was translated according to EORTC guidelines into English, French, and Italian. For patients with PNH and AA until now, the standard assessment for QoL was to use the EORTC Quality of Life Questionnaire (QLQ-C30) or the Functional Assessment of Chronic Illness Therapy Fatigue Instrument (FACIT-Fatigue). We herewith present a new instrument aimed to be better tailored to the needs of PNH and AA patients. The anticipated fourth development phase will be performed for psychometric validation; however, we already explored the internal consistency of the hypothesised scales and found the results to be very good. Hence, the new QLQ-AA/PNH with 54 items can be used in trials and clinical studies from now on, according to EORTC strategy even if the scoring algorithm at this point is preliminary and the QLQ-AA/PNH might change slightly after phase IV. This is important, as there are no other disease-specific instruments available for AA/PNH patients right now.
S Wheelwright - One of the best experts on this subject based on the ideXlab platform.
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International validation of the EORTC QLQ-ELD14 questionnaire for assessment of health-related quality of life elderly patients with cancer
British Journal of Cancer, 2013Co-Authors: S Wheelwright, A-s Darlington, D Fitzsimmons, P Fayers, J I Arraras, F Bonnetain, E Brain, A Bredart, W-c Chie, J GiesingerAbstract:Background: Older people represent the majority of cancer patients but their specific needs are often ignored in the development of health-related quality of life (HRQOL) instruments. The European Organisation for Research and Treatment of Cancer (EORTC) QLQ-ELD15 was developed to supplement the EORTC’s core questionnaire, the QLQ-C30, for measuring HRQOL in patients aged >70 years in oncology studies. Methods: Patients ( n =518) from 10 countries completed the QLQ-C30, QLQ-ELD15 and a Debriefing Interview. Eighty two clinically stable patients repeated the questionnaires 1 week later (test–retest analysis) and 107 others, with an expected change in clinical status, repeated the questionnaires 3 months later (response to change analysis, RCA). Results: Information from the Debriefing Interview, factor analysis and item response theory analysis resulted in the removal of one item (QLQ-ELD15→QLQ-ELD14) and revision of the proposed scale structure to five scales (mobility, worries about others, future worries, maintaining purpose and illness burden) and two single items (joint stiffness and family support). Convergent validity was good. In known-group comparisons, the QLQ-ELD14 differentiated between patients with different disease stage, treatment intention, number of comorbidities, performance status and geriatric screening scores. Test–retest and RCA analyses were equivocal. Conclusion: The QLQ-ELD14 is a validated HRQOL questionnaire for cancer patients aged ⩾70 years. Changes in elderly patients’ self-reported HRQOL may be related to both cancer evolution and non-clinical events.
Binita M. Kamath - One of the best experts on this subject based on the ideXlab platform.
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Development of a Novel Tool to Assess the Impact of Itching in Pediatric Cholestasis
The Patient - Patient-Centered Outcomes Research, 2018Co-Authors: Binita M. Kamath, Linda Abetz-webb, Ciara Kennedy, Bonnie Hepburn, Martha Gauthier, Nathan Johnson, Sharon Medendorp, Alejandro Dorenbaum, Lora Todorova, Benjamin L. ShneiderAbstract:Objectives The aim was to develop a clinical outcome assessment (COA) for itching in children with cholestatic pruritus. Methods This prospective study aimed to enroll patients aged 4–30 years with Alagille syndrome (ALGS) or progressive familial intrahepatic cholestasis type 1 and caregivers of patients aged 5 months to 14 years. Eligible patients experienced itching during ≥3 of the 7 days before enrollment and had not undergone liver transplant or surgical interruption of the enterohepatic circulation. Open-ended qualitative Interviews confirmed that itching was a primary concern for patients and caregivers. Diaries were modified and then evaluated by participants during cognitive Debriefing. Interview results were reviewed by clinical, COA and statistical experts. Diary questions were revised following an interim analysis before finalizing the Itch Reported Outcome (ItchRO). Results Thirty-six Interviews were analyzed, representing 25 families of patients with ALGS. Itching was reported spontaneously (without prompting by the Interviewer) by ten of 12 patients with ALGS and 19 of 20 caregivers. Consequences of itching included skin damage (78%), mood changes (59%), and difficulties staying asleep (59%) or falling asleep (53%). Two versions of the ItchRO were developed: ItchRO(Patient) for self-completion by patients and ItchRO(Observer) for caregivers. The ItchRO diaries comprise a single scorable item to assess itch and are to be completed twice daily (morning and evening). Conclusions Itching was the most bothersome ALGS symptom reported by study participants. We have developed the ItchRO(Patient) and ItchRO(Observer) to assess itching in children with ALGS and other cholestatic liver diseases. These diaries are being validated for use in clinical trials.
