ALBERT

Description: Introduction: Iron chelation therapy (ICT) is essential in removing excess iron deposited in body organs, ultimately preventing organ failure and extending the lives of patients (pts) with transfusion-dependent hematological disorders such as β-thalassemia and myelodysplastic syndromes (MDS). As a life-long treatment, traditional ICT (deferoxamine, Desferal®, DFO) is based on a burdensome regimen (subcutaneous delivery 5–7 times a week) that has been shown to negatively impact on pts’ health-related quality of life (HRQoL). The oral chelator deferasirox (Exjade®) is less burdensome to pts offering 24-hour ICT, 7 days a week. Methods: This substudy was part of a single arm, multicenter, 1-year open-label trial (the EPIC study) to investigate the efficacy/safety of deferasirox. The first 558 pts with a variety of hematological disorders were recruited. These pts came from sites in seven countries: Australia, Belgium, France, Germany, UK, Greece, and Italy. Treatment-naïve pts and those having previously received ICT (DFO or deferiprone [Ferriprox®] exclusively, or combined) participated (n=558). Pts were asked at baseline, week 4 and week 52 (end of study [EOS]) to complete the 36-item Short Form health survey (SF-36). The SF-36 is a self-administered questionnaire and measures eight HRQoL domains: physical functioning; role-physical; bodily pain; general health; vitality; social functioning; role-emotional; and mental health. Mean change in SF-36 domain scores were calculated for all pts who had completed data at baseline and week 4, as well all those with completed data at baseline and EOS. All domains are scored so that higher scores indicate a better QoL. Results: Overall, the mean age of the 558 pts (274 β-thalassemia, 168 MDS, 50 sickle cell disease and 66 other anemias) recruited to take part in this substudy was 40.8 years (SD=22.58); 51.5% of patients (n=289) were male and 48.5% (n=272) were female. Within this sample, 337 pts aged ≥16 years completed the SF-36 at baseline, 322 at week 4 and 277 at EOS. Mean domain scores for pts at baseline, week 4 and EOS are presented in Table 1. With the exception of role-emotional (mean=0.78, SD=40.56), mean change in SF-36 domain scores significantly improved (P

Description: Abstract 4763 Introduction: Hemophilia A is a rare but serious bleeding disorder caused by a deficiency in blood clotting factor VIII (FVIII). To examine the impact of hemophilia A and replacement FVIII therapies from a humanistic and economic perspective, a targeted literature review was conducted. Methods: Searches were conducted in MEDLINE® and the National Health Service Economic Evaluation Database, using combinations of disease, patient-reported outcome and economic-related key words, limited to articles published in English between January 2000 and January 2010 (inclusive). A total of 34 full-text articles (from 653 abstracts retrieved) were selected for detailed consideration. Results: Findings revealed increased mortality rates and decreased life expectancy among people with hemophilia A, compared with the general population. This is largely a result of the transmission of blood-borne viruses (e.g. HIV and Hepatitis-C) due to use of plasma-derived FVIII (pdFVIII) concentrates. Improvements in viral attenuation processes plus donor screening practices for pdFVIII products have reduced the risks of viral transmission, but risk of transmission still exists in relation to non-enveloped viruses and other unknown pathogens. Newly developed recombinant FVIII therapies minimizes these risks; however, such therapies are not currently widely available globally. All available FVIII therapies use demanding regimens requiring regular time-consuming injections, which can be detrimental to patients’ health-related quality of life and limit adherence to replacement FVIII therapy. Non-adherence to factor replacement therapy is associated with diminished product efficacy, poorer health outcomes and increasing economic expenditure. Treating hemophilia A is costly, primarily due to the high acquisition cost of replacement FVIII products. However, indirect costs related to patient disability and level of care also contribute to economic burden. Prophylactic treatment initially may appear to be more expensive than on-demand treatment but is associated with greater clinical efficacy and improved long-term outcomes, which may lead to cost savings over the course of a patient's lifetime. Conclusions: Hemophilia A is associated with considerable humanistic and economic burden. There remain substantial unmet needs with regard to the safety, convenience, global access to treatment incl. reliable product supply and total care costs associated with factor replacement therapies. Disclosures: Strandberg-Larsen: Novo Nordisk A/S: Employment. Gater:Novo Nordisk A/S: Research Funding. Marlow:Novo Nordisk A/S: Research Funding. Thomson:Novo Nordisk A/S: Research Funding.

Description: Abstract 2486 Poster Board II-463 Introduction: Iron chelation therapy (ICT) is essential in removing excess iron deposited in body organs, ultimately preventing organ failure and extending the lives of patients (pts) with transfusion-dependent hematological disorders such as β-thalassemia. Conventional ICT (deferoxamine, Desferal®, DFO) requires a burdensome regimen (subcutaneous delivery 5–7 times a week) that has been shown to negatively impact pts' health-related quality of life (HRQL) and adherence to therapy. Adherence to ICT affects survival and therefore a well-tolerated and effective ICT regimen is required. The once-daily oral chelator, deferasirox (Exjade®) offers 24-hour ICT, 7 days a week and is potentially less burdensome to pts. Methods: As part of a larger single-arm, multicenter, open-label trial investigating the efficacy and safety of deferasirox (EPIC study), 217 β-thalassemia pts 〉16 years of age were included in this subanalysis. Findings reported here investigate differences in satisfaction, adherence and HRQoL following treatment with deferasirox, in β-thalassemia patients with prior experience of DFO monotherapy, or DFO in combination with deferiprone (Ferriprox®, L1). All pts were asked at baseline and week 52 (end of study [EOS]) to complete the 19-item Satisfaction with ICT questionnaire (SICT) and the 36-item Short Form health survey (SF-36). Mean change scores between baseline and EOS on the four domains of the SICT (Perceived Effectiveness of ICT, Side Effects of ICT, Acceptance of ICT, and Burden of ICT) and the eight domains (Physical Functioning, Role-Physical, Bodily Pain, General Health, Vitality, Social Functioning, Role-Emotional, and Mental Health) and two summary scores (Physical and Mental Component Scores) of the SF-36 were calculated for patients who completed the respective instruments at both timepoints. Higher scores on the SICT and SF-36 represent greater HRQL, satisfaction with, and adherence to treatment. Adherence to deferasirox was assessed by two SICT items asking pts how often they thought about stopping their ICT, and how often they took their ICT exactly as directed by their doctor. Results: 217 β-thalassemia pts were recruited (mean age 30.6 ± 7.7); 42.9% (n = 93) male, 57.1% (n = 124) female. Of 216 pts with prior history of ICT, 62.0% (n=134) had prior DFO monotherapy and 37.0% (n=80) had prior DFO in combination with L1 (2 patients had prior LI monotherapy; not included in this analysis due to the small patient number). SICT and SF-36 mean-change domain scores were based on patients who completed questionnaires at baseline and EOS (SICT: 105 DFO and 46 DFO+L1 pts; SF-36: 90 DFO and 42 DFO+L1 pts). Significant improvements (p

Description: INTRODUCTION: Acute myeloid leukemia (AML) is a rare hematologic cancer primarily affecting older people, with a median age at diagnosis of 67 years old (Almeida & Ramos, 2016). Heterogeneity in the presentation, functional status and presence of comorbidities among AML patients presents unique challenges for treatment. Intensive chemotherapy (IC), although the best option for prolonging survival, carries a risk of early death and other trade-offs, including significant time spent in the hospital. The toxicity of IC treatment and the requirement for prolonged hospitalizations may negatively affect patients' physical functioning and health-related quality of life (HRQoL). Novel, low-intensity treatments can be administered in the clinic and may pose less risk of immediate toxicities but may be associated with reduced efficacy. Each patient and family will approach these trade-offs differently, yet few studies examine the process of shared decision-making in AML. We aimed to better understand this process by eliciting patient/family and physician narratives about expectations and attitudes towards AML treatments. METHODS: Ten physicians in the US (n=4), UK (n=3) and Canada (n=3) and 12 AML patients (all US) and a member of their family took part in an individual, 60-minute qualitative telephone interview. Further interviews are scheduled and any additional data at the time of presentation will also be reported. The interviews followed a semi-structured guide comprising open-ended questions. The overarching aim of the interviews was to understand the value of living longer for AML patients who are not candidates for standard IC and to explore the treatment decision process from the patient, family and physician perspective. During the interviews, each participant (patient, family member or physician) completed a rating exercise in which they were asked to rate a list of pre-defined factors (9-10 factors) on a scale of 0 (not at all important) to 3 (very important) to determine their importance in AML treatment decisions. As well as providing a numerical rating for each factor, each participant was asked why they selected their rating and which three factors they would consider the most important. RESULTS: Across all three groups, relief in AML symptoms (namely fatigue and pain), longer survival and better QoL were equally considered the three most important factors when making a treatment decision. All three groups described the interaction between QoL and longer survival, explaining that any increase in survival would be important, but only if QoL (time spent with family, maintaining hobbies/interests) was maintained or improved. Physician's advice was also important to AML patients when making decisions regarding treatment. Other treatment-related factors within the rating task were rated as very important for at least a subset of patients, with no factors widely considered to be of limited importance. All participants noted that while relief of AML symptoms, longer survival and quality of life were all important, these had to be considered in light of treatment side effects and risk of infection. Likelihood of being hospitalized was important to family members, as they wanted to spend quality time at home with their loved ones, while patients and physicians considered hospitalization as an inevitable consequence of the disease and treatment. All participants reported that AML patients would consider taking any form of treatment if suitable and effective, regardless of the mode of administration. However, it was acknowledged that oral treatments would be more convenient and less invasive. The relative importance of location of care/treatment delivery was influenced by proximity to resources and whether the participant was based in an urban or rural setting. The ability to receive treatment at home was considered beneficial. CONCLUSIONS: The results from this rating exercise and qualitative interviews showed convergence across all stakeholders, indicating that AML patients, family and physicians have similar priorities regarding treatment decisions, prioritizing symptom relief, survival, and quality of life. The predominant treatment pathways in AML each require trade-offs in these factors, demonstrating the importance of shared decision making in ensuring the most appropriate treatment is selected for a given individual, in accordance with their values, goals, and preferences. Figure Disclosures LeBlanc: Jazz Pharmaceuticals: Research Funding; Daiichi-Sankyo: Membership on an entity's Board of Directors or advisory committees; Otsuka: Consultancy, Membership on an entity's Board of Directors or advisory committees; Heron: Membership on an entity's Board of Directors or advisory committees; Helsinn: Consultancy; Flatiron: Consultancy; NINR/NIH: Research Funding; Duke University: Research Funding; Astra Zeneca: Consultancy, Research Funding; Amgen: Membership on an entity's Board of Directors or advisory committees; Medtronic: Membership on an entity's Board of Directors or advisory committees; Seattle Genetics: Consultancy, Research Funding; Celgene: Honoraria; AbbVie: Membership on an entity's Board of Directors or advisory committees; Pfizer Inc: Consultancy; American Cancer Society: Research Funding; Agios: Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; CareVive: Consultancy. Walter:Seattle Genetics: Research Funding; Agios: Consultancy; Amgen: Consultancy; Amphivena Therapeutics: Consultancy, Equity Ownership; Aptevo Therapeutics: Consultancy, Research Funding; Argenx BVBA: Consultancy; Astellas: Consultancy; BioLineRx: Consultancy; BiVictriX: Consultancy; Boehringer Ingelheim: Consultancy; Boston Biomedical: Consultancy; Covagen: Consultancy; Daiichi Sankyo: Consultancy; Jazz Pharmaceuticals: Consultancy; Kite Pharma: Consultancy; New Link Genetics: Consultancy; Pfizer: Consultancy, Research Funding; Race Oncology: Consultancy. Hernandez-Aldama:Pfizer Inc: Consultancy. Sully:Pfizer Inc: Consultancy; Adelphi Values Ltd: Employment. Bell:Pfizer Inc.: Employment, Equity Ownership. Johnson:Pfizer Inc: Consultancy; Adelphi Values Ltd: Employment. Peloquin:Pfizer Inc: Employment, Equity Ownership. Gater:Pfizer Inc: Consultancy; Adelphi Values Ltd: Employment. Welch:Pfizer Inc: Employment, Equity Ownership. O'Hara:Adelphi Values Ltd: Employment; Pfizer Inc: Consultancy. Russell:Astellas: Consultancy, Honoraria, Speakers Bureau; Pfizer Inc: Consultancy, Honoraria, Speakers Bureau; Jazz: Consultancy, Honoraria, Speakers Bureau; DSI: Consultancy, Honoraria, Speakers Bureau. Horikoshi:Pfizer Inc: Consultancy. Maze:Pfizer Inc: Consultancy; Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees.

Description: Introduction: Tyrosine kinase inhibitors (TKIs) represent the standard of care for patients with chronic-phase chronic myeloid leukemia (CP-CML). The emergence and availability of second-generation TKIs (dasatinib, nilotinib and bosutinib) has led to increased choice for patients and physicians, with data from randomized clinical trials indicating improved efficacy of such therapies compared to the first-generation TKI (imatinib). Differences in the efficacy of second-generation TKIs are negligible; however, all have distinct dosing instructions and safety profiles. Understanding the patient perspective is invaluable for optimizing healthcare decisions, with interest in information regarding patient preferences growing among a wide variety of stakeholders, including regulatory agencies, reimbursement bodies and prescribers. However, evidence is lacking regarding treatment preferences and priorities for patients with CP-CML. The aim of this study was to explore attributes of currently-licensed TKI therapies that are relevant and important to patients with CP-CML. This research represents the first step in the conduct of a discrete choice experiment (DCE), or conjoint analysis, to explore treatment preferences more broadly among CP-CML patients. Patient-guided design is crucial to ensuring attributes of investigation are meaningful. Hence the qualitative insights will advise and validate attribute and level selection for inclusion in the forthcoming DCE. Methods: In-depth qualitative interviews were conducted with 12 US-based patients with a clinician-confirmed diagnosis of CP-CML. The sample included 10 females and 2 males (median age 46 years) who were diagnosed 2 years (n=3), 4-7 years (n=5), 10-12 years (n=3) and 16.5 years (n=1) prior to interview. Ten patients had received a second-generation TKI. Current treatments were dasatinib (n=8), bosutinib (n=1) and imatinib (n=3). Previous treatments included imatinib (n=6), nilotinib (n=3) and dasatinib (n=1). The interviews utilized concept elicitation techniques to capture attributes of importance in both TKI treatment experience and TKI treatment selection. Transcripts were thematically analyzed using Atlas.ti. Results: Open-ended questioning elicited an array of attributes related to TKI treatments. It was important to all patients to ensure that a CP-CML treatment was effective. Treatment efficacy was conveyed with multiple descriptors; most commonly "BCR-ABL" (n=7) or major molecular response "MMR", polymerase chain reaction "PCR" and "remission" (each n=3). Eight patients discussed the importance of dose frequency, specifically the burden of scheduling self-administration of oral tablets (n=5). Seven patients conveyed the difficulty of taking a TKI with specific meal requirements, most notably when fasting is required prior to and post-administration (n=4). A multitude of important treatment side effects were elicited at investigation into the patient TKI treatment experience. Fatigue was reported by 11 patients (n=10 spontaneous) and considered by most patients (n=8/11) to be directly attributable to treatment versus the CML itself. Four patients considered fatigue an inevitable component of TKI treatment. Nausea was elicited by 7 patients (n=5 spontaneous) and some patients (n=5/9) considered it to be influential in treatment selection. Muscle pain was reported by 6 patients (n=4 spontaneous), while skin rash was reported by 5 patients and bone pain by 4 patients (all spontaneous). Diarrhea was reported by 4 patients (n=3 spontaneous). Additional symptoms reported by ≤2 patients are presented in Figure 1. Spontaneously elicited concepts were ranked by importance. All patients considered efficacy within the top three most important attributes. Six patients considered fatigue to be either of second or third most-importance. The rankings in Figure 2 serve to portray the varied perspectives and experiences of patients on TKI treatments. Conclusions: The TKI treatment experience for CP-CML patients is multifaceted. This type of study - the first that we know of - supplies valuable insight into the patient perspectives of TKI treatment to establish a foundation to better inform relevant decisions in the field. The qualitative findings will provide the input necessary to inform attribute and level selection to characterize treatment profiles ahead of an upcoming patient-preference DCE. Disclosures Mason: Adelphi Values: Employment; Pfizer: Consultancy. Mamolo:Pfizer: Employment, Equity Ownership. Johnson:Pfizer Inc: Consultancy; Adelphi Values Ltd: Employment. Viqueira:Pfizer Inc: Employment, Equity Ownership. Gater:Pfizer Inc: Consultancy; Adelphi Values Ltd: Employment. Russell-Smith:Pfizer: Employment, Equity Ownership. Tatlock:Adelphi Values: Employment. Shah:Pfizer: Employment, Equity Ownership. Cappelleri:Pfizer: Employment, Equity Ownership.

Description: INTRODUCTION: Patient experience data provides an opportunity to explore patient's perspectives on current and potential treatments. Gaining patient narratives on the expectation, tolerance and attitudes towards treatments can enhance clinical management and inform discussions with payers and regulatory agencies. Patient experience data is particularly beneficial in conditions where there is no clear, prescribed treatment pathway as it can contextualize joint treatment decisions made by patients and their treating physicians. Life expectancy among elderly patients with hematogical cancers such as acute myeloid leukemia (AML) is short. Patients with these conditions may be eligible for intensive chemotherapy (IC) or may face a decision between non-intensive chemotherapy (NIC) or palliative care. Further, eligible patients may not wish to undergo the taxing treatment regimens associated with IC. A targeted literature review was conducted to understand the trade-offs associated with treatment decision making and to gain patient perspectives on the value of extended overall survival (OS) in hematological cancers. METHODS: Searches of computerized bibliographic databases including PubMed (Medline), EMBASE and PsycINFO were conducted using the OVID platform. Searches were conducted using a combination of keywords and Medical Subject Headings (MeSH) terms. To be selected for review, articles must have contained keywords in the title and/or abstract and include information regarding patient experience or perspectives. Articles focussed on children (