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Introduction Acute 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. Methods Participants 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. Results The 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%). Conclusion Although 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. Funding Astellas Pharma Inc., Northbrook, IL, USA. Electronic supplementary material The online version of this article (doi:10.1007/s40487-016-0029-8) contains supplementary material, which is available to authorized users.

Haptoglobin (Hp) genotype 2-2 increases cardiovascular diabetes complications. In type 2 diabetes, α-tocopherol was shown to lower cardiovascular risk in Hp 2-2, potentially through HDL function improvements. Similar type 1 diabetes data are lacking. We conducted a randomized crossover pilot of α-tocopherol supplementation on HDL function [i.e., cholesterol efflux (CE) and HDL-associated lipid peroxides (LP)] and lipoprotein subfractions in type 1 diabetes. Hp genotype was assessed in members of two Allegheny County, PA, type 1 diabetes registries and the CACTI cohort; 30 were randomly selected within Hp genotype, and 28 Hp 1-1, 31 Hp 2-1 and 30 Hp 2-2 were allocated to daily α-tocopherol or placebo for 8 weeks with a 4-week washout. Baseline CE decreased with the number of Hp 2 alleles (p-trend = 0.003). There were no differences in LP or lipoprotein subfractions. In intention-to-treat analysis stratified by Hp, α-tocopherol increased CE in Hp 2-2 (β = 0.79, p = 0.03) and LP in Hp 1 allele carriers (β Hp 1-1 = 0.18, p = 0.05; β Hp 2-1 = 0.21, p = 0.07); reduced HDL particle size (β = −0.07, p = 0.03) in Hp 1-1 carriers; increased LDL particle concentration in Hp 1-1; and decreased it in Hp 2-2 carriers. However, no significant interactions were observed by Hp. In this type 1 diabetes study, HDL function worsened with the number of Hp 2 alleles. α-Tocopherol improved HDL function in Hp 2-2 carriers and appeared to adversely affect lipid peroxides and lipoprotein subfractions among Hp 1 allele carriers. As no significant interactions were observed, findings require replication in larger studies.

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.