BACKGROUND: Desmoid tumors (or aggressive fibromatosis) are locally infiltrative connective-tissue tumors that can arise in any anatomic location; they can be asymptomatic, or they can result in pain, deformity, swelling, and loss of mobility and/or threaten visceral organs with bowel perforation, hydronephrosis, neurovascular damage, and other complications. Existing clinical trial endpoints such as the Response Evaluation Criteria in Solid Tumors (version 1.1) and progression-free survival are inadequate in capturing treatment efficacy. This study was designed to develop a novel clinical trial endpoint by capturing patient-reported outcomes (PROs). METHODS: Following best practices in qualitative methodology, this study used concept elicitation (CE) interviews to explore desmoid patients' perspectives on key disease-related symptoms and impacts. Qualitative analysis was performed to determine the relative frequency and disturbance of symptoms and impacts as well as other characteristics of these concepts. A draft PRO scale was then developed and tested with cognitive interviewing. Information from the interviews was subsequently incorporated into the refined PRO scale. RESULTS: CE interviews with desmoid patients (n = 31) helped to identify salient concepts and led to a draft scale that included symptom and impact scales. Cognitive interviews were completed with additional patients (n = 15) across 3 phases. Patient input was used to refine instructions, revise and/or remove items, and modify the response scale. This resulted in an 11-item symptom scale and a 17-item impact scale. CONCLUSIONS: This is the first disease-specific PRO instrument developed for desmoid tumors. The instrument is available as an exploratory endpoint in clinical trials. This study highlights the feasibility and challenges of developing PRO instruments for rare diseases.
IntroductionAcute myeloid leukemia (AML) imposes significant burden on patients, their families, and the healthcare system. Published literature has reported many AML signs and symptoms, as well as their impact on patients. However, there are no publications on the experience of living with AML from the patient's perspective. In this study, we performed qualitative interviews with patients with AML to understand their experience.MethodsParticipants were recruited from the US and Japan. All patients were screened to assess eligibility, and were divided into four subgroups (i.e., newly-diagnosed, high-intensity chemotherapy; newly-diagnosed, low-intensity chemotherapy; relapse/refractory; and post-transplant). Patients were interviewed over the phone by a trained researcher and asked about their day-to-day experience with AML. Signs/symptoms and impacts were coded, analyzed using Atlas.ti software, and reported as frequencies, with the medians of patient-reported disturbance levels (0–10) computed for each symptom and impact.ResultsThe most commonly reported sign/symptom in the US was fatigue (95.7%), followed by bruising and weakness (both 78.3%), and in Japan, nausea (94.4%), followed by fatigue and headache (both 88.9%). The most commonly reported impact in the US was a decreased ability to maintain social/familial roles (91.3%), followed by anxiety and a decreased ability to function (both 87.0%), and most commonly reported in Japan was anxiety, a decreased ability to function, and remission uncertainty (94.4%).ConclusionAlthough the frequency of signs/symptoms and their level of disturbance varied between the US and Japan, there was remarkable consistency in the types of signs/symptoms and impacts reported across all patients. The consistency in the experience of the disease across patients suggests that measurement of AML experience can be achieved by using the same tool for most, if not all, of these patients.FundingAstellas Pharma Inc., Northbrook, IL, USA.Electronic supplementary materialThe online version of this article (doi:10.1007/s40487-016-0029-8) contains supplementary material, which is available to authorized users.
11022 Background: Desmoid tumors (DT) are locally aggressive and cause significant morbidity. Clinical trials in DT typically utilize response rates and progression free survival as primary endpoints. However, these endpoints do not capture improvements in clinical symptoms. To date, there are no validated PRO tools in DT to capture the patient experience and efficacy of a drug. Methods: A review of the published literature and interviews with sarcoma clinicians were used to formulate a list of signs and symptoms and impact on patients (pts). These were collected to build a conceptual model. DT pts (n = 31) with a range of anatomical locations and presentations were interviewed, initially in an open-ended fashion, followed by interrogating the conceptual model. For the concepts that pts reported, they were asked to rate how disturbing each was on a 0-10 scale (0 being not at all, and 10 being as bad as they can imagine). The pts interview data was then used to refine the conceptual model and generate two new PRO instruments Results: Pt interviews demonstrated that across tumor locations, the most frequent and disturbing symptoms were: ‘muscle’ pain (65% pts, median disturbance (MD) of 6.8), ‘nerve’ pain (73%, MD 6.0), and fatigue (65%, MD 5.0). Some symptoms were specific to tumor locations, especially abdominal tumors. Restricted range of motion (68%, MD 4.0), fear (84%, MD 6.5), sleep disturbance (77%, MD 7.5) , disfigurement (81%, MD 6.8), and impact on daily activities (65%, MD 6.8) were the most frequent and disturbing impact on pts lives. These concepts were then used to develop two new PRO instruments: the sign and symptom PRO includes 11 items; the impact on pts lives instrument includes 17 items. The instruments vary in asking pts about the last 24 hours, or the last week. Conclusions: This is the first validated PRO tool in DT. This tool adequately captures symptoms central to the DT pts experience and its impacts on their lives. The instruments are ready for implementation in a DT clinical trial for further evaluation of their measurement properties.
INTRODUCTION: One way to capture the patient reality of living with disease is to talk with patients and listen to them describe their experience. We sought to understand the patient perspective regarding the signs and symptoms of acute myeloid leukemia (AML) and the impact of these on patients, through direct, qualitative patient interviews. METHODS: We conducted individual interviews with patients with AML (median age=48, 37% male) in Japan and the USA following a semi-structured interview guide, containing both open- and closed- ended questions. These questions aimed to understand the patient's experience both spontaneously and with specific probing. Patients were asked to describe their individual experience of living with AML including signs, symptoms, impacts, and the experience during treatment of the disease. RESULTS: Forty-one patients were interviewed in the two regions: 23 in US, and 18 in Japan. The sign, symptom, and impact 'concepts' identified were very similar across regions. Forty-nine symptoms were mentioned by US patients; 32 by Japanese patients. The signs/symptoms reported most frequently and as most bothersome included: fatigue, bruising, weakness, dizziness, shortness of breath, and bleeding (Table 1). Twenty-three unique impacts of signs/symptoms were mentioned in the US and 20 in Japan. The most frequent and bothersome impacts reported included: decreased ability to maintain social/familial roles, anxiety, decreased ability to function, depression, fear, remission uncertainty, appetite loss, and low sex drive (Table 1). These concepts were corroborated by two additional sources: interviews with clinicians and published literature. DISCUSSION: The priority concepts expressed by patients were relevant and important to all four AML patient groups in both regions. This data provides valuable insight into patients' experiences with AML and helps to select the appropriate patient-reported outcomes for clinical trials. Table 1. AML Symptom and Impact Frequencies US Frequency (N=23), n (%) Japan Frequency (N=18), n (%) Symptom Fatigue 22 (95.6) 16 (88.8) Bruising 18 (78.2) 7 (38.8) Weakness 18 (78.2) 15 (65.2) Dizziness 15 (65.2) 12 (52.1) Shortness of Breath 13 (56.5) 15 (65.2) Bleeding 12 (52.1) 13 (72.2) Impact Decreased ability to maintain social/familial roles 21 (91.3) 13 (72.2) Anxiety 20 (86.9) 17 (94.4) Decreased ability to function 20 (86.9) 17 (94.4) Depression 16 (69.5) 7 (38.8) Fear 15 (65.2) 12 (52.1) Remission uncertainty 15 (65.2) 17 (94.4) Appetite loss 14 (60.8) 13 (72.2) Low sex drive 11 (47.8) 7 (38.8) Disclosures Tomaszewski: Astellas Pharma, Inc.: Consultancy. Fickley:Astellas Pharma, Inc.: Consultancy. Maddux:Astellas Pharma, Inc.: Consultancy. Krupnick:Astellas Pharma, Inc.: Consultancy. Bahceci:Astellas Pharma Global Development: Employment. Paty:Astellas Pharma, Inc.: Consultancy. van Nooten:Astellas: Employment.
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