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Aim:To determine measurement equivalence of paper and electronic application of the hematological malignancy-patient-reported outcome (HM-PRO), a specific measure for the evaluation of patient-reported outcomes in HMs. Patients & methods:Following International Society of Pharmacoeconomics and Outcomes Research ePRO Good Research Practice Task Force guidelines, a total of 193 adult patients with different HMs were recruited into a multicenter prospective study. The paper and the electronic version of the instrument were completed in the outpatient clinics in a randomized crossover design with a 30 min time interval to minimize the learning effect. Those who completed the paper version first, completed the electronic version after 30 min and vice versa. Instrument version and order effects were tested on total score of the two parts of the HM-PRO (Part A: quality of life and Part B: signs & symptoms) in a two-way ANOVA with patients as random effects. Intraclass correlation coefficients (95 CI) and Spearman's rank correlation coefficients were used to evaluate test–retest reliability and reproducibility. The effects of instrument version and order were tested on total score of the two parts of HM-PRO. Results:The questionnaire version and administration order effects were not significant at the 5 level. There were no interactions found between these two factors for HM-PRO (Part A [quality of life]; p  0.95); and (part B [signs and symptoms]; p  0.72]. Spearman's rank correlation coefficients were greater than 0.9, and intraclass correlation coefficients ranged from 0.94 to 0.98; furthermore, the scores were not statistically different between the two versions, showing acceptable reliability indexes. Noteworthy, the difference between the completion time for both paper (mean 6:38 min) and electronic version (mean 7:29 min) was not statistically significant (n  100; p  0.11). Patients did not report any difficulty in completing the electronic version during cognitive interviews and were able to understand and respond spontaneously. Conclusion:Measurement equivalence has been demonstrated for the paper and electronic application of the HM-PRO.

Incorporating patient-reported outcomes (PROs) when studying patients with immune thrombocytopenia (ITP) is essential since treatment decisions are complex and using platelet count only partly explains disease burden. Since most symptoms are only experienced subjectively and are seldom captured during clinician-based evaluations, using self-report is crucial for early symptom detection. However, capturing the patient’s illness experience necessitates using well-developed and validated instruments. This article provides insight on the importance of using PROs in ITP, summarizes the methodological steps to develop PRO instruments, and discusses challenges related to integrating PROs into research and clinical practice.

The goal of treatment of relapsed/refractory multiple myeloma (RRMM) is to control the disease, prolong survival, reduce disease-related symptoms, and improve quality of life (QoL). Comprehensive evaluation of new treatment regimens in RRMM pts is worthwhile. We aimed to evaluate QoL, treatment satisfaction, response to treatment and safety during Ixazomib-Lenalidomide-Dexamethasone (IRd) treatment as ≥ 2nd line in RRMM pts in a real world setting.

Background

Ionova: BMS: Research Funding; Takeda: Research Funding. Vinogradova:Novartis: Honoraria, Research Funding.

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.

Pemberton-Whiteley: CML Advocates Network: Membership on an entity's Board of Directors or advisory committees; Patient Evidence: Equity Ownership; AbbVie: Other: Grant funding; Amgen: Consultancy, Other: Grant funding, Speakers Bureau; Bristol-Myers Squibb: Consultancy, Other: Grant funding, Speakers Bureau; Celgene: Consultancy, Other: Grant funding; Acute Leukemia Advocates Network (ALAN): Consultancy; Pfizer: Consultancy, Other: Grant Funding, Speakers Bureau; Takeda: Other: Grant funding; Shire: Other: Grant Funding; Kyowa Kirin: Other: Grant funding; Novartis: Consultancy, Other: Grant funding, Speakers Bureau; Incyte: Consultancy, Other: Grant funding; Jazz: Other: Grant funding, Speakers Bureau; Janssen: Consultancy, Other: Grant funding; Daiichi Sankyo: Other: Grant funding; Gilead: Other: Grant funding, Speakers Bureau; Leukaemia Care: Employment. Oliva:Celgene Corporation: Consultancy, Honoraria, Speakers Bureau; Novartis: Consultancy, Speakers Bureau; Apellis: Consultancy. Geissler:Incyte: Research Funding; Roche: Consultancy; Novartis: Consultancy, Research Funding, Speakers Bureau; Janssen: Consultancy, Speakers Bureau; UCB: Consultancy, Speakers Bureau; Servier: Consultancy; Takeda: Research Funding; Pfizer: Consultancy, Research Funding, Speakers Bureau; Biomarin: Consultancy; Amgen: Consultancy; Bristol-Myers Squibb: Consultancy, Research Funding, Speakers Bureau. Wintrich:Takeda: Consultancy; Janssen: Other: Grant Funding; Celgene: Other: Grant Funding; Novartis: Consultancy, Other: Grant Funding. Salek:Pfizer: Honoraria, Speakers Bureau; Merck: Consultancy; Agios Pharmaceuticals, Inc.: Consultancy, Honoraria. Ionova:Takeda, BMS: Other: Principal Investigator of IISR, Research Funding. Tate:Quality Health: Employment. Bradley:Quality Health: Employment.

Introduction

Ionova: Takeda, BMS: Other: Principal Investigator of IISR, Research Funding. Chelysheva:Novartis: Consultancy, Honoraria; Fusion Pharma: Consultancy. Shukhov:Novartis: Consultancy; Pfizer: Consultancy. Turkina:Novartis: Consultancy, Speakers Bureau; Pfizer: Consultancy; Bristol Myers Squibb: Consultancy; fusion pharma: Consultancy; Novartis: Consultancy, Speakers Bureau.

Background

Ionova: Takeda, BMS: Other: Principal Investigator of IISR, Research Funding. Baryakh:Takeda: Consultancy, Honoraria, Speakers Bureau.

There is a continued unmet medical need in pts with relapsed/refractory Hodgkin's lymphoma (RR HL). Curing HL pts who have refractory disease after salvage chemotherapy, who relapse after ASCT, or those who are not candidates for ASCT, remains a clinical challenge due to limited effective treatments. There are data available indicating that brentuximab vedotin (BV) brings considerable promise for the treatment of pts with RR HL. Information about BV treatment effectiveness and tolerability both from physician's and patient's perspective is worthwhile in this difficult patient population. We aimed to evaluate clinical and patient-reported outcomes in RR HL patients receiving BV as >2ndtreatment line. Here we report the outcomes with respect to clinical response, tolerability, quality of life (QoL) and symptoms after 3 mos of BV treatment.

Oliva: Sanofi: Consultancy, Speakers Bureau; La Jolla: Consultancy; Amgen: Consultancy, Speakers Bureau; Celgene: Consultancy, Other: Royalties, Speakers Bureau; Janssen: Consultancy, Speakers Bureau; Novartis: Consultancy, Speakers Bureau. Ionova:BMS: Research Funding; Takeda: Research Funding.

Oliva: La Jolla: Consultancy; Janssen: Consultancy, Speakers Bureau; Amgen: Consultancy, Speakers Bureau; Novartis: Consultancy, Speakers Bureau; Celgene: Consultancy, Other: Royalties, Speakers Bureau; Sanofi: Consultancy, Speakers Bureau. Ionova:Takeda: Research Funding; BMS: Research Funding.

Oliva: Sanofi: Consultancy, Speakers Bureau; Celgene: Consultancy, Other: Royalties, Speakers Bureau; La Jolla: Consultancy; Amgen: Consultancy, Speakers Bureau; Novartis: Consultancy, Speakers Bureau; Janssen: Consultancy, Speakers Bureau. Ionova:Takeda: Research Funding; BMS: Research Funding.

Background:Patient reported outcome (PRO) assessments, which include quality of life (QoL) and symptoms, have become increasingly important, aiding in identifying and informing patient needs. Patients with acute myeloid leukemia (AML) have poor clinical prognosis and outcomes. There is insufficient knowledge about the impact of disease and treatment on AML patients' lives.

Background:The responsiveness of a patient-reported outcome (PRO) measure is an important measurement property which is required to show that the instrument is capable of detecting change over time or after a therapeutic intervention. Minimal clinically important difference (MCID) on the other hand is the smallest change in the scores that is meaningful from a patient's perspective. Evaluating both responsiveness and MCID of a newly developed PRO instrument is a measurement necessity. A novel hematological malignancy (HM) specific PRO tool, HM-PRO, has been developed for use in daily clinical practice as well as clinical research. The HM-PRO is a composite measure consisting of two scales: Part A-measuring the impact on patients' quality of life (QoL); and Part B-measuring the effect of signs and symptoms experienced by the patients. The aims of this study were to evaluate the responsiveness and estimate the MCID of the newly developed HM-PRO.

Introduction:Patient-reported outcome (PRO) measures not only have been widely used in clinical research but also increasingly employed in daily clinical practice to understand the health outcomes of medical interventions. A novel hematological malignancy (HM) specific PRO tool, HM-PRO, has been recently developed for use in daily clinical practice. The HM-PRO is a composite measure consisting of two scales: Part A - measuring the ‘impact on patients’ quality of life (QoL); and Part B-measuring the effect of ‘signs and symptoms’ experienced by the patients. Both scales have linear scoring system ranging from 0 to 100, with higher scores representing greater impact on QoL and symptom burden. The assessment of the “meaningfulness” of HM-PRO scores is essential if clinicians are to be able to use the instrument to understand patient health outcomes to aid their clinical decision-making and encourage better patient engagement. One way of enhancing the clinical utility of scores on multi-item questionnaires is by investigating the importance (to patients and clinicians) of cross-sectional differences by anchoring those differences and changes to clinically familiar events that are related to patient well-being. The aims of the present study were to determine the relationship between the HM-PRO scores and a Global Question (GQ) measuring the impact on a patient's life from patients' perspective and to identify bands of HM-PRO scores equivalent to each GQ descriptor, reflecting patients' global rating of PROs.

Background:Patient with hematological malignancies (HM) often experience worsening of quality of life (QoL) due to both the disease and its treatments. Impaired QoL may have negative impact on treatment outcomes leading to change in treatment decision-making. However, this has not been captured in a systematic manner in routine clinical practice and there is no specific instrument for such purpose. Thus, there is the need of a new patient-reported outcome (PRO) measure. The aims of this study were to identify issues important to patients with HM and to develop a new PRO measure for use in daily clinical practice.

Ionova: BMS: Research Funding; MSD: Speakers Bureau. Bulieva:BMS: Research Funding. Vinogradova:BMS: Research Funding. Kurbatova:BMS: Research Funding. Nikitina:BMS: Research Funding. Novitskaya:BMS: Research Funding. Rodionova:BMS: Research Funding. Usacheva:BMS: Research Funding. Chukavina:BMS: Research Funding. Shumkova:BMS: Research Funding.

Ionova: MSD: Speakers Bureau; BMS: Research Funding. Nikitina:BMS: Research Funding.

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.

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.

Dasatinib as a front-line is a promising treatment option for CP-CML pts. Assessment of benefits and risks of this treatment regimen both from physician's and patient's perspective sounds worthy. We aimed to study QoL and clinical outcomes of dasatinib as a front-line treatment in CP CML pts in a real world setting.

Clinicians frequently underestimate quality of life (QoL), level of depression, and the severity of important disease- and treatment-related symptoms in patients with haematological malignancies, while overestimating other aspects of disease. Better symptom control may result in improved treatment outcomes in this patients population. Adequate symptom assessment in routine clinical practice is very helpful to provide patient-centred care. The goal of the study was to assess symptom burden in adult patients with different haematological malignancies, namely malignant lymphomas (ML), multiple myeloma (MM) and chronic myeloid leukemia (CML), and identify similarities and differences in symptom burden across these tumor entities.

No relevant conflicts of interest to declare.

Myelofibrosis (MF) is the most symptomatic of the myeloproliferative neoplasms and is associated with the greatest symptom burden and poorest prognosis. Patient-reported outcomes is an effective way to identify patients' needs and risks/benefits of MF treatment. We aimed to study quality of life (QoL) and symptom burden in MF patients in a real-world setting.

No relevant conflicts of interest to declare.

Ionova: BMS: Research Funding. Nikitina:BMS: Research Funding. Fedorenko:BMS: Research Funding. Gritsenko:BMS: Research Funding. Ivanova:BMS: Research Funding. Kuchma:BMS: Research Funding. Shnaider:BMS: Research Funding. Sannikova:BMS: Research Funding. Usacheva:BMS: Research Funding. Kurbatova:BMS: Research Funding.

Information about the efficacy and safety of the second-line therapy with dasatinib in patients in chronic phase chronic myeloid leukemia (CML-CP) at long-term follow-up is limited. Evaluation of benefits and risks of the treatment in a “real-world” study both from physician’s and patient’s perspective is worthwhile to better define treatment outcomes in this patients’ population. We aimed to study clinical and patient-reported outcomes as well as safety of the second-line therapy by dasatinib in CML-CP patients with imatinib resistance or intolerance treatment at long-term follow-up.

Ionova: BMS: Research Funding. Nikitina:BMS: Research Funding. Gritsenko:BMS: Research Funding. Ivanova:BMS: Research Funding. Kuchma:BMS: Research Funding. Shnaider:BMS: Research Funding. Sannikova:BMS: Research Funding. Fedorenko:BMS: Research Funding. Kurbatova:BMS: Research Funding.

There is limited published data about the efficacy and safety of the second-line therapy with dasatinib in patients in chronic phase chronic myeloid leukemia (CML-CP) in a “real world” patients setting outside clinical trials. In addition, comprehensive evaluation of benefits and risks of the treatment is worthwhile to better define treatment outcomes in this patients' population. We aimed to study clinical and patient-reported outcomes as well as safety of dasatinib treatment in a “real world” setting within the context of its approved indication through the analysis of prospectively collected data in patients with imatinib resistance or intolerance receiving dasatinib as the second-line therapy.

Abstract 4445

No relevant conflicts of interest to declare.

No relevant conflicts of interest to declare.

No relevant conflicts of interest to declare.

No relevant conflicts of interest to declare.

Abstract 4757

Abstract 372

Abstract 2561

During the last decade immunoablative therapy with ASCT (IT+ASCT) has been used with increasing frequency as a therapeutic option for MS patients. Among a number of unanswered questions is the timing of IT+ASCT. We have identified 3 strategies of IT+ASCT depending on the stage in disease process: early, conventional and salvage/late. The goal of our research was to study clinical and patient-reported outcomes in MS patients after early, conventional and salvage/late transplantation. 54 patients with MS (secondary progressive - 26 patients, primary progressive -10, progressive-relapsing - 1, and relapsing-remitting - 17) from 6 medical centers were included in this study (mean age - 32.0, range: 17–51; male/female - 21/33). 13 patients underwent early, 37 patients - conventional, and 4 patients - salvage/late transplantation. Median EDSS at base-line was 6.0 (range 1.5 – 8.0). The median follow-up duration was 18 months (range 6 – 84 months). Neurological and quality of life (QoL) evaluation was performed at baseline, at discharge, at 3, 6, 9, 12 months, and every 6 months thereafter following IT+ASCT. MRI examinations were conducted at baseline, at 6, 12 months, and at the end of follow-up. FACT-BMT and FAMS were used for QoL evaluation. Notably, no transplant-related deaths or unpredictable severe adverse events were observed. All 42 patients (6 - early transplantation; 32 - conventional transplantation; 4 - salvage/late transplantation) with the follow-up of longer than 9 months experienced clinical stabilization or improvement. More than half of them (27 patients) improved: 8 patients showed significant improvement in EDSS, 9 patients improved by 1.0 point, and 10 - by 0.5 points on EDSS. Notably, all of the patients after early transplantation (mean age - 28.0) improved at least by 0.5 points on EDSS. 15 patients achieved stabilization. 2 patients deteriorated to a worse score after 18 months of stabilization; 2 others progressed after 12 and 30 months of improvement, respectively. All of the patients with clinical stabilization and improvement had negative MRI scans. Out of 24 patients included in QoL analysis 22 exhibited improved QoL 6 months post-transplantation. In conclusion, IT+ASCT appears to be an effective treatment for MS both in terms of clinical and patient-reported outcomes. The data obtained point to feasibility of early, conventional and salvage/late transplantation in MS patients. Our data support the hypothesis that transplantation is more effective in young patients at early stages of rapidly progressing disease. Further studies should be done to establish the best timing for transplantation and to validate IT+ASCT regimens in patients receiving early, conventional and salvage transplantation.

During the last decade immunoablative therapy with ASCT (IT+ASCT) has been used with increasing frequency as a therapeutic option for MS patients. Among a number of unanswered questions is the timing of IT+ASCT. We have identified 3 strategies of IT+ASCT depending on the stage in disease process: early, conventional and salvage/late. The goal of our research was to study clinical and patient-reported outcomes in MS patients after early, conventional and salvage/late transplantation. 54 patients with MS (secondary progressive - 26 patients, primary progressive -10, progressive-relapsing - 1, and relapsing-remitting - 17) from 6 medical centers were included in this study (mean age - 32.0, range: 17–51; male/female - 21/33). 13 patients underwent early, 37 patients - conventional, and 4 patients - salvage/late transplantation. Median EDSS at base-line was 6.0 (range 1.5 – 8.0). The median follow-up duration was 18 months (range 6 – 84 months). Neurological and quality of life (QoL) evaluation was performed at baseline, at discharge, at 3, 6, 9, 12 months, and every 6 months thereafter following IT+ASCT. MRI examinations were conducted at baseline, at 6, 12 months, and at the end of follow-up. FACT-BMT and FAMS were used for QoL evaluation. Notably, no transplant-related deaths or unpredictable severe adverse events were observed. All 42 patients (6 - early transplantation; 32 - conventional transplantation; 4 - salvage/late transplantation) with the follow-up of longer than 9 months experienced clinical stabilization or improvement. More than half of them (27 patients) improved: 8 patients showed significant improvement in EDSS, 9 patients improved by 1.0 point, and 10 - by 0.5 points on EDSS. Notably, all of the patients after early transplantation (mean age - 28.0) improved at least by 0.5 points on EDSS. 15 patients achieved stabilization. 2 patients deteriorated to a worse score after 18 months of stabilization; 2 others progressed after 12 and 30 months of improvement, respectively. All of the patients with clinical stabilization and improvement had negative MRI scans. Out of 24 patients included in QoL analysis 22 exhibited improved QoL 6 months post-transplantation. In conclusion, IT+ASCT appears to be an effective treatment for MS both in terms of clinical and patient-reported outcomes. The data obtained point to feasibility of early, conventional and salvage/late transplantation in MS patients. Our data support the hypothesis that transplantation is more effective in young patients at early stages of rapidly progressing disease. Further studies should be done to establish the best timing for transplantation and to validate IT+ASCT regimens in patients receiving early, conventional and salvage transplantation.