Purpose Pyruvate kinase deficiency (PKD) is a rare disease and understanding of its epidemiology and associated burden remains limited. With no current curative therapy, clinical manifestations can be life threatening, clinically managed by maintaining adequate hemoglobin levels through transfusion and subsequent support, but with frequent complications. Treatment goals are to maintain/improve the patient’s quality of life. With new therapies, reliable, valid, and relevant patient-reported outcome (PRO) tools are required for use in clinical trials. Methods Systematic literature search identified no current PRO tools for capturing/measuring the impact of PKD and treatments in clinical trials. Therefore, the search strategy was revised to consider conditions analogous to PKD in terms of symptoms and impacts that might serve as parallels to the experience in PKD; this included sickle cell anemia, thalassemia, and hemolytic anemia. Psychometric properties, strengths, and weakness of selected appropriate PRO instruments were compared, and recommendations made for choice of PRO tools. Results In adult populations, EORTC QLQ C30 and SF-36v2 are recommended, the former being a basic minimum, covering generic HRQoL, and core symptoms such as fatigue. In pediatric populations, PedsQL Generic Core Scale to measure HRQoL and PedsQL MFS scale to measure fatigue are recommended. Conclusions Some symptoms/life impacts may be unique to PKD and not observable in analogous conditions. A ‘Physico-Psychosocial Model’ derived from the ‘Medical Model’ is proposed to form the basis for a hypothesized conceptual framework to address the development of PKD-specific PRO instruments. Electronic supplementary material The online version of this article (10.1007/s11136-018-2025-y) contains supplementary material, which is available to authorized users.
Background: Our aim was to identify health-related quality-of-life (HRQoL) issues and symptoms in patients with haematological malignancies (HMs) and develop a conceptual framework to reflect the inter-relation between them. Methods: A total of 129 patients with HMs were interviewed in a UK multicentre qualitative study. All interviews were audio recorded, transcribed and analysed using NVivo-11. Results: Overall, 34 issues were reported by patients and were grouped into two parts: quality of life (QoL) and symptoms. The most prevalent HRQoL issues were: eating and drinking habits; social life; physical activity; sleep; and psychological well-being. Furthermore, most prevalent disease-related symptoms were: tiredness; feeling unwell; breathlessness; lack of energy; and back pain. The most prevalent treatment side effects were: tiredness; feeling sick; disturbance in sense of taste; and breathlessness. Conclusions: Both HMs and their treatments have a significant impact on patients’ HRQoL, in particular on issues such as job-role change, body image and impact on finances.
Aims: The impact of haematological malignancies (HM) on patients' health-related quality of life (HRQoL) is still not well understood. The aim of this study was to identify HRQoL issues and symptoms in patients with HM to be included in a new patient-reported outcome measure for use in routine clinical practice. Methods: In a multicentre observational study carried out in the UK, adult patients with various HM, capable of reading English and give written informed consent were recruited from five hospitals in England and Wales. This qualitative study employed semi-structured face-to-face interviews with open-ended questions related to the impact of haematological malignancy and its treatment on HRQoL and symptoms. All the interviews were audio recorded and transcribed verbatim. Content analysis was carried out using the NVivo 11 qualitative analysis software. The themes and the sub-themes generated from the transcribed interviews were discussed during a 2-day "data definition" panel meeting by 2 hematologists, 1 patient research partner, 1 representative of a hematology patient organisation and 3 QoL research experts to select items for inclusion in the prototype instrument. These items will be further re-grouped and refined using cognitive debriefing, content validity and factor analysis. Results: 127 (male=75; mean age = 61.6 years; SD=15.1; median age 65.4 years; and age range =18-88 years) with mean duration of the HM of 3.7 years (SD=4.3; median=2.1 years; and range= 19 days-23 years) were recruited into the study. Diagnoses were: Acute Myeloid Leukaemia (18); Acute Lymphoid Leukaemia (7); Chronic Myeloid Leukaemia (12); Chronic Lymphatic Leukaemia (11); Aggressive Non-Hodgkin Lymphoma (16); Indolent Non-Hodgkin Lymphoma (14); Hodgkin Lymphoma (10); Multiple Myeloma (21); Myeloproliferative Neoplasm (10); and Myelodysplastic Syndrome (8). 383 items were reported by the patients under different themes and subthemes. 117 of these items were reported by more than 5% of the patients. 149 items were selected by the data definition panel to be included in the prototype instrument. The most prevalent QoL issues important to HM patients (Figure 1) were: 'eating and drinking habits (57 patients changed eating and drinking habits; 48 reported loss of appetite; 29 stopped drinking alcohol; and 11 reported increase in appetite); impaired social life and participatory function (86); impaired physical ability or independency (71); disturbed sleep (66); impaired psychological well-being (64); impaired daily activities (61); impaired ability to go on holidays or travelling (60); impaired work life & studies (57 ); impaired sexual life (55); impaired ability to manage finances (34); recreational activities and pastime (32); and relationships (26), from high to low prevalence, respectively. With respect to disease related symptoms, 102 issues were identified, the most prevalent being 'tiredness (65), feeling unwell (28), breathlessness (24), lack of energy (21), back pain (17) and weight loss (17)", from high to low prevalence respectively. Out of 124 treatment related symptoms identified, the most prevalent were: 'tiredness (73); feeling sick (36); lack of energy (20); taste disturbance (20); breathlessness (15); and diarrhoea (15)', from high to low prevalence respectively. Conclusion: The findings of the qualitative and item generation phase clearly indicate that HMs affect patients' QoL significantly. However, in the absence of a validated measure for use in routine clinical practice, this is not captured in a systematic manner. Thus, this highlights the need for the development and validation of a new HM-specific PRO measure for use in such settings. Psychometric testing of the prototype instrument will be carried out to establish the measurement properties of the new HM-specific PRO measure. Figure Figure. Disclosures Salek: EHA: Other: Educational and travel grant. Fielding:Amgen: Membership on an entity's Board of Directors or advisory committees; Baxalta: Membership on an entity's Board of Directors or advisory committees; Pfizer: Membership on an entity's Board of Directors or advisory committees. Kell:Celgene: Honoraria; Novartis: Honoraria; Sunesis: Equity Ownership. Oliva:Pharma Companies: Honoraria; Italy: Speakers Bureau; Pharma Companies: Consultancy. Ionova:BMS: Research Funding; MSD: Other: lecturer bureau.
Background: The quality of life of patients at all stages of hematological malignancy is greatly affected by the disease and its treatment. There is a wide range of healthrelated quality of life (HRQoL) issues important to these patients. Any new instrument developed to measure HRQoL of such patients should be content valid, i.e., the items should be comprehensively relevant to the patients and their health condition. The aim of the present study was to examine content validity of a hematological malignancy specific patient reported outcome measure (HM-PRO) developed for use in routine clinical practice. Methods: Following literature review and semi-structured interviews, the generated themes and sub-themes were discussed to develop the prototype version of the HM-PRO. A 4-step approach was used for content validation: initial testing and cognitive interviewing; item rating; content validity panel meeting; final field testing and cognitive interviewing. Additional questions related to patients' perception of recall period and preferred sentence structure (i.e., question or statement) of the items were also asked during cognitive interviews. Results: The content analysis of 129 transcribed semi-structured interviews resulted in the prototype version of the instrument consisting of 58 items grouped into two parts: Part A (impact/HRQoL-34 items) and Part B (signs and symptoms-24 items). The initial testing showed intra-class correlation coefficient (ICC) of >0.8 for both Part A and Part B. Item rating for language clarity, completeness, relevance, and response scale by experts and patients showed content validity index for scales average >0.8 for both Part A and Part B, except 0.64 for relevance for Part A by the patient panel. The final testing of the revised version of the instrument showed the Cronbach's alpha value of
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