Real-world evidence data on the monoclonal antibody erenumab in migraine prevention: perspectives of treating physicians in Germany

Abstract Background Erenumab, the first-in-class fully human monoclonal antibody targeting the calcitonin gene-related peptide receptor, was shown to be efficacious and safe for the prophylactic treatment of migraine in adults in randomized clinical trials. Large-scale, real-world evidence in multi-centre settings is still needed to confirm these results. Erenumab patient profiles outside clinical trials and physicians’ treatment patterns, as well as data from patients treated in Germany, a severely impacted population, are not published yet. Methods TELESCOPE was a multi-centre survey gathering real-world data from 45 German headache centres between July 2019 and December 2019. The project consisted of two parts. In the first part, treating physicians shared their experiences on current erenumab treatment with regard to patient profiles, treatment patterns and treatment responses. In the second part, a retrospective chart review was conducted of 542 migraine patients treated with erenumab for at least three months. Treatment responses focused on various aspects of patients’ quality of life. Results The analysis of 542 patients’ charts revealed that three-month treatment with erenumab significantly reduced monthly headaches, migraine and acute medication days. Furthermore, headache intensity and frequency were reduced in over 75 % and accompanying aura in 35 % of patients. The clinical global impression scale revealed a general improvement in 91 % of patients. According to the treating physicians’ professional judgement, 83 % of patients responded to erenumab and 80 % were satisfied with the treatment. Physicians evaluated restricted quality of life, the number of monthly migraine days and previous, prophylactic treatments as the main components of the current patient profile for monoclonal antibody recipients. Based on the assessment of physicians, erenumab reduced migraine symptoms in 65 % and increased quality of life in more than 75 % of their patients. Conclusions TELESCOPE confirms positive treatment responses with erenumab shown in clinical trials in a real-world multi-centre setting. The results show consistently positive experiences of physicians utilizing erenumab in clinical practice and underline that therapy with this monoclonal antibody is effective in migraine patients, particular in those, who have failed several prophylactic therapies.

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Real-world Evidence Data on the Monoclonal Antibody Erenumab in Migraine Prevention: Perspectives of Treating Physicians in Germany

10.21203/rs.3.rs-631442/v1 ◽

2021 ◽

Author(s):

Andreas Straube ◽

Philipp Stude ◽

Charly Gaul ◽

Katrin Schuh ◽

Mirja Annika Koch

Keyword(s):

Quality Of Life ◽

Monoclonal Antibody ◽

Clinical Trials ◽

Real World ◽

Treatment Patterns ◽

Patient Profile ◽

Real World Evidence ◽

Patient Profiles ◽

Treatment Responses

Abstract Background Erenumab, the first-in-class fully human monoclonal antibody targeting the calcitonin gene-related peptide receptor, was shown to be efficacious and safe for the prophylactic treatment of migraine in adults in randomized clinical trials. Large-scale, real-world evidence in multi-centre settings is still needed to confirm these results. Erenumab patient profiles outside clinical trials and physicians’ treatment patterns, as well as data from patients treated in Germany, a severely impacted population, are not published yet. MethodsTELESCOPE was a multi-centre survey gathering real-world data from 45 German headache centres between July 2019 and December 2019. The project consisted of two parts. In the first part, treating physicians shared their experiences on current erenumab treatment with regard to patient profiles, treatment patterns and treatment responses. In the second part, a retrospective chart review was conducted of 542 migraine patients treated with erenumab for at least three months. Treatment responses focused on various aspects of patients’ quality of life. ResultsThe analysis of 542 patients’ charts revealed that three-month treatment with erenumab significantly reduced monthly headaches, migraine and acute medication days. Furthermore, headache intensity and frequency were reduced in over 75 % and accompanying aura in 35 % of patients. The clinical global impression scale revealed a general improvement in 91 % of patients. According to the treating physicians’ professional judgement, 83 % of patients responded to erenumab and 80 % were satisfied with the treatment. Physicians evaluated restricted quality of life, the number of monthly migraine days and previous, prophylactic treatments as the main components of the current patient profile for monoclonal antibody recipients. Based on the assessment of physicians, erenumab reduced migraine symptoms in 65 % and increased quality of life in more than 75 % of their patients. ConclusionsTELESCOPE confirms positive treatment responses with erenumab shown in clinical trials in a real-world multi-centre setting. The results show consistently positive experiences of physicians utilizing erenumab in clinical practice and underline that therapy with this monoclonal antibody is effective in migraine patients, particular in those, who have failed several prophylactic therapies.

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In search of a gold standard patient-reported outcome measure to use in the evaluation and treatment-decision making in migraine prevention. A real-world evidence study

The Journal of Headache and Pain ◽

10.1186/s10194-021-01366-9 ◽

2021 ◽

Vol 22 (1) ◽

Author(s):

Alicia Alpuente ◽

Victor J Gallardo ◽

Edoardo Caronna ◽

Marta Torres-Ferrus ◽

Patricia Pozo-Rosich

Keyword(s):

Quality Of Life ◽

Clinical Trials ◽

Real World ◽

Medical Decision ◽

Stepwise Logistic Regression ◽

Primary Efficacy ◽

Patient’S Perspective ◽

Real World Evidence ◽

Patient Reported

Abstract Background Patient-Reported Outcomes (PROs) have been developed to numerically quantify disability, impact and quality of life. They have been widely used in migraine clinical trials. However, we still do not know which PRO more accurately reflects preventive treatment response from a patient’s perspective or which one may help us with treatment decisions in clinical practice. They have been used to enforce the efficacy results in clinical trials and real-world evidence so far. The aim of this study was to analyze which PROM is (1) better correlated with all primary efficacy endpoints and (2) which one is better associated with treatment continuation with CGRP-mAbs at week-12, which is usually the moment when this decision is made. Methods Patients with migraine who had received 3 administrations of CGRP-mAbs were evaluated in this prospective cohort study. Primary efficacy outcomes considered: a change in migraine days (MMD), headache days (MHD), pain intensity (INT), acute medication days (AMD) and 50% responder rate. The Spearman coefficient (rs) was the measure used for quantify the strength of the correlation between PROMs and treatment efficacy outcomes changes. A stepwise logistic regression identified which PROM was independently associated with treatment continuation at week-12. Results 263 patients completed 12 weeks of treatment. The efficacy outcomes and PROMs scores were statistically significantly reduced at week-12 for all patients. The role function-restrictive (RFR) domain of the Migraine-Specific Quality of Life (MSQ) questionnaire was statistically significantly correlated with all primary efficacy outcomes. Relative changes in MSQ total score (OR[95%]: 0.840[0.619-0.973]; p=0.037) and Patient Global Impression of Change (PGIC) scale (OR[95%]: 15.569[6.254-31.533]; p<0.001) were the PROMs associated with treatment continuation as independent factors at week-12. Conclusions Changes in MSQ questionnaire and PGIC scale at week-12 were the PROMs with higher association with CGRP-mAbs response from a patient’s perspective and medical decision-taking.

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A Real-World Evidence Systematic Literature Review of Health-Related Quality of Life, Costs and Economic Evaluations in Newly-Diagnosed Multiple Myeloma Patients

Blood ◽

10.1182/blood.v128.22.4777.4777 ◽

2016 ◽

Vol 128 (22) ◽

pp. 4777-4777

Author(s):

Candice Yong ◽

Ellen Korol ◽

Zaeem Khan ◽

Huamao Mark Lin ◽

Juliette Thompson ◽

...

Keyword(s):

Quality Of Life ◽

Clinical Trials ◽

Real World ◽

Indirect Costs ◽

Newly Diagnosed ◽

Financial Difficulties ◽

Real World Evidence ◽

Related Quality ◽

Health Related

Abstract Introduction Current treatment for multiple myeloma (MM) is not curative, but aims to provide patients with improved disease control to extend survival. Newly diagnosed MM (NDMM) can have significant implications on the health-related quality of life (HRQoL) of a patient and increase the economic burden to both the patient and healthcare system. Strict protocols followed for clinical trials mean results seen in clinical trials may not reflect routine clinical practice. To assess the burden of illness of NDMM, systematic reviews of real-world humanistic and economic data was conducted. Methods Two systematic literature reviews (SLRs) of the humanistic and economic burden in NDMM were conducted, covering the published literature from Jan 2007 to June 2016. Data sources reviewed included Medline, Embase, PubMed (for e-publications ahead of print), and select hematology, oncology and health services research conferences. Eligibility criteria were limited to NDMM patients receiving first-line treatment (including induction, stem cell transplant [SCT], consolidation, and maintenance therapy) reported in observational studies or cost effectiveness analysis (CEA). Results Eleven studies met the inclusion criteria for the HRQoL SLR and 29 studies for the economic SLR. The study populations were heterogeneous in terms of patient and clinical characteristics (Table 1). A summary of HRQoL measures and baseline values are shown in Table 2. Cancer or myeloma specific tools were used to assess HRQoL in 5 studies; 2 studies utilized a generic HRQoL tool (EQ-5D and SF-36). Symptoms of interest associated with MM and MM treatment - either pain, fatigue, or neuropathy - were assessed in 8 of the included studies. The humanistic burden was pronounced in patients with NDMM. Across studies, HRQoL was poor at diagnosis; the European Organization for Research and treatment of Cancer Quality of Life Questionnaire (EORTC-QLQ-C30) global scores ranged from 55 to 64 at diagnosis (scale from 0-100 with higher values indicating better HRQoL), and improved as patients received therapy. Domains related to physical attributes, including symptoms, were more adversely affected than those related to mental domains. Pain and fatigue were consistently highlighted as the most problematic symptoms throughout the treatment period; however, both symptoms improved over the course of therapy. Neuropathy was measured in 3 studies, each in selected populations; patients who were ineligible for SCT reported greater burden due to neuropathy, and neuropathy increased over the course of first-line treatment. Financial difficulties also contributed to impairment on HRQoL for patients throughout the measured period. Of the 29 studies identified as eligible for the economic review, 22 included either direct or indirect costs, and 7 studies were CEA; 11 studies included estimates of real-world resource use. Costs varied across studies, mainly due to different cost inputs considered and currencies reported. Overall, direct costs ranged from 7,534 USD 2014 per patient per month (unadjusted medical and pharmacy costs for 1st line lenalidomide treatment) to 213,166 USD 2014 per patient (direct costs associated with autologous SCT and one year follow-up). The primary cost drivers of direct medical costs were largely medical care, including hospitalizations and ambulatory care. Two studies reported out-of-pocket expenses, ranging from 3,478 USD 2013 (annual cost to patient in 1st year after diagnoses) to 4,666 USD (year not reported; per treatment episode for 1st year after treatment initiation). The results from the CEA studies differed significantly (Table 3). Conclusions MM specific HRQoL measures indicate reduced HRQoL at diagnosis which gradually improves throughout the treatment period in newly diagnosed patients (before they experience relapse). Pain, fatigue and financial difficulties were domains reported as problematic to patients. Resource use burden studies primarily reported in- and out-patient visits. Indirect costs were reported to be as high as 4,666 USD, which further supports the financial difficulties reported in the HRQoL studies. The burden of NDMM for a patient is physical, emotional, and financial. Real-world evidence on HRQoL and economic outcomes reported from the patient perspective is complementary to evidence from clinical trials and provides an insight into the burden of NDMM. Disclosures Yong: Takeda: Employment. Korol:ICON plc: Employment. Khan:ICON plc: Employment. Lin:Takeda: Employment. Thompson:Takeda: Consultancy. Valovicova:Takeda Pharmaceutical Company Ltd: Consultancy. Luptakova:Takeda Oncology: Employment. Seal:Millennium Pharmaceuticals, Inc., a wholly owned subsidiary of Takeda Pharmaceutical Company Limited: Employment, Equity Ownership.

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Comprehensive Geriatric Assessment in Consecutive Newly-Diagnosed Elderly Multiple Myeloma Patients in China: A Multicentered, Prospective, Non-Interventional Study

Blood ◽

10.1182/blood-2019-125447 ◽

2019 ◽

Vol 134 (Supplement_1) ◽

pp. 5493-5493

Author(s):

Yuan Yao ◽

Dehui Zou ◽

Aijun Liao ◽

Xiaoxia Chu ◽

Wei Wang ◽

...

Keyword(s):

Quality Of Life ◽

Multiple Myeloma ◽

Clinical Trials ◽

Cognitive Impairment ◽

Geriatric Assessment ◽

Real World ◽

Comprehensive Geriatric Assessment ◽

Newly Diagnosed ◽

Frail Patients

Background: Multiple Myeloma (MM) is a disease of the elderly, whose prognoses are highly heterogeneous. Hence International Myeloma Working Group (IMWG) proposed geriatric assessment (GA) in 2015, including daily activity and comorbidity status, to better discriminate between fit and frail patients (Palumbo et al, 2015). However, IMWG recruited patients from clinical trials instead of real world practices. Therefore we studied GA in elderly MM patients consecutively in China, along with other perspectives which are known to be problematic in elderly population that were previously left unnoticed, such as nutrition status, risk of cognitive impairment, risk of depression, and quality of life. Aim: Our study centers on the feasibility to perform a more comprehensive geriatric assessment (cGA) in elderly MM patients, current cGA status in elderly MM patients in China, and the cGA difference between Chinese patients and patients in the IMWG study. Method: From August 2017 to April 2019, we continuously recruited 336 newly diagnosed elderly (age ≥ 65) MM patients from 21 centers in China. cGA was performed at diagnosis, after treatment cycle 1, after cycle 4, and 1 year after treatment. cGA includes physical conditions (ECOG), activities of daily living (ADL), instrumental ADL (IADL), mini-nutritional assessment (MNA-SF), geriatric depression scale (GDS), mini-mental state examination (MMSE), quality of life (SF-36) and Charlson comorbidity index (CCI). Staging was assessed at baseline (International Staging System (ISS) & Revised ISS) and hematological responses were evaluated along with each cGA timepoint. Results: We pool-analyzed data of 336 newly-diagnosed elderly MM patients. The median age was 70 (range 65-88) and 25.5% of patients were older than 75 years. 336 (100%) patients were able to complete cGA, and median assessment time was 40 minutes (range 20-70). Upon diagnosis, only 34% and 37.5% of patients had full ADL and IADL respectively. 38.5% of patients had moderate to high risk of depression (GDS ≥ 6). 13.2% of patients were malnourished (MNA-SF ≤ 7), while 46.3% of patients were at risk of malnutrition (8 ≤ MNA-SF ≤ 11). 41% of patients had at least one comorbidity (CCI ≥ 1). 45.7% of patients had moderate to intermediate risk of cognitive impairment (MMSE ≤ 26). Grouping by IMWG-GA index, our study identified 59.9% patients in frail group (vs 39% in IMWG study), 15.8% in intermediate (vs 31% in IMWG) and 24.3% in fit (vs 30% in IMWG). 69% of patients received proteasome inhibitor-containing regimens and 20.7% of patients received lenalidomide-containing regimens. Best hematological responses in fit and intermediate groups were better than responses in frail group (≥ PR rate: 88.5% in fit, 94.4% in intermediate vs 77.5% in frail). Median follow up time was 10 months. To date, 215 (64%) patients have finished the cGA after cycle 1; 164 (48.8%) patients have finished the cGA after cycle 4; 91 (27.1%) patients has finished all 4 planned cGA and improvements in cGA were observed in the majority of these patients. Conclusion: Our study showed significant CGA heterogeneity in elderly MM patients. Even in the IMWG-GA "fit" group, nutrition, depression and cognitive impairment remain problems. Frail patients took up a larger proportion in Chinese elderly MM patients compared to IMWG study. Our study strongly justifies the necessity for cGA in elderly patients with MM, more so in the real world MM patients than in the clinical trials. Disclosures No relevant conflicts of interest to declare.

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PRO64 UNDERSTANDING REAL-WORLD TREATMENT PATTERNS AND QUALITY OF LIFE IN PATIENTS WITH PEMPHIGUS AND PEMPHIGOID USING A PATIENT REGISTRY DATABASE

Value in Health ◽

10.1016/j.jval.2019.04.1697 ◽

2019 ◽

Vol 22 ◽

pp. S347

Author(s):

M. Yale ◽

M. Bucci ◽

V. Boulanger ◽

A. Seebald ◽

S. Rossov ◽

...

Keyword(s):

Quality Of Life ◽

Real World ◽

Treatment Patterns ◽

Patient Registry ◽

Registry Database

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Targeted Literature Review of Patient Reported Outcomes (PROs) in Chronic Myeloid Leukemia (CML) Patients Receiving Second and Later Lines of Treatment

Blood ◽

10.1182/blood-2020-140402 ◽

2020 ◽

Vol 136 (Supplement 1) ◽

pp. 26-27

Author(s):

Shiyu Zhang ◽

Rodrigo Maegawa ◽

Savita Nandal ◽

Pallavi Patwardhan

Keyword(s):

Quality Of Life ◽

Clinical Trials ◽

Chronic Myeloid Leukemia ◽

Real World ◽

Myeloid Leukemia ◽

Observational Studies ◽

Symptom Burden ◽

Pharmaceutical Corporation ◽

Line Of Therapy

Background: Tyrosine kinase inhibitors (TKIs) have revolutionized the treatment of chronic CML in the last several years; however, the failure rates associated with TKIs still remain high in second line (50%) and later lines (75%-80%) treatment. Also, patients often experience adverse events associated with these TKIs, but are often left with no alternative treatment in real world practice which might result in TKI cycling. There is a need to understand the quality of life (QoL) and symptom burden among these patients, in order to better manage their disease and also to explore new treatments. The objectives of this study were to summarize the PROs among patients with CML receiving 2nd/3rd/4th line (2L/3L/4L) treatment, and the instruments used for QoL and symptom burden assessment in clinical trials and real world studies. Methods: Embase, MEDLINE, and the Cochrane Library were searched using pre-defined search terms to identify studies published from 1/1/2010 to 6/15/2020. Following the title/abstract and full-text screening based on the inclusion/exclusion criteria (see Table), one reviewer independently extracted all relevant data with random quality check (~20% of the sample) by a 2nd reviewer. Included studies reported QoL, symptom burden, or any PROs of adult CML patients who received 2L/3L/4L treatment. Only clinical trials and observational studies published in English were included, including conference abstracts. Results: Database search identified 1,377 records, which were screened to identify 20 studies (5 observational studies and 15 clinical trial-based studies) that met eligibility criteria (see Figure). Of the included studies, 16 targeted chronic phase CML patients only. More than half of the studies (n=14) focused on patients receiving 2L treatment (bosutinib, dasatinib, or nilotinib) only, and six reported QoL data on patients receiving 2L/3L/4L treatment, out of which four studies reported results by line of therapy. Out of 15 studies reporting a follow-up period, 13 observed patients ≥ 12 months with a maximum of 24 months. The duration between observations were usually 12 weeks for observational studies; while shorter duration was observed in clinical trials. Six clinical trial-based studies were associated with one trial (NCT00261846). Instruments used to assess QoL included four generic instruments [Functional Assessment of Cancer Therapy - General (FACT-G), EuroQol 5 dimension (EQ-5D), 36-Item Short Form Survey (SF-36), European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30)], three disease specific instruments [Functional Assessment of Cancer Therapy - Leukemia (FACT-Leu), MD Anderson Symptom Inventory chronic myeloid leukemia (MDASI-CML), Comprehensive Symptom Profile in Chronic Myeloid Leukemia Patients (CSP Leuk-CML)], one Work Productivity and Activity Impairment (WPAI) questionnaire, and two additional general questions on QoL. Ten studies applied both generic and disease specific instruments altogether to evaluate patients' QoL and symptom burden. For 2L TKI treatment, studies have shown improvement in QoL following treatment; however, one study indicated that although patients were content with their QoL, they reported poor symptom scores on mobility, usual activities, and pain/discomfort. Among four studies examining PROs by line of therapy, only one study reported results on the 4L cohort individually, which showed lowest baseline FACT-G and FACT-Leu score in the 4L cohort compared to other cohorts, especially in terms of the physical and emotional well-being domain. Discrepancies existed in QoL between 2L- and 3L cohorts. Two studies found QoL to be similar between the 2L- and 3L cohorts, while the other two observed the QoL was lower in the 3L cohorts. In addition, three reported that QoL scores remained stable throughout observation period regardless of the line of therapy, but one study demonstrated the utility score got improved in the 2L but not the 3L cohort. Conclusions: Evidence on PROs in CML patients receiving 3L/4L treatment is very limited, especially real-world data. Existing research indicates patients with CML receiving later lines of treatment may have poor quality of life. Robust real-world studies with longer follow-up period are needed to provide evidence on later lines treatment management among CML patients. Disclosures Zhang: Novartis Pharmaceutical Corporation: Current Employment. Maegawa:Novartis Pharmaceutical Corporation: Current Employment, Current equity holder in publicly-traded company. Patwardhan:Novartis Pharmaceutical Corporation: Current Employment, Current equity holder in publicly-traded company.

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Quality of Life Effects of an Oral Fixed Combination of Netupitant and Palonosetron in Chemotherapy-Induced Nausea and Vomiting Prevention: Real-World Evidence in Patients with Breast Cancer Receiving Anthracycline-Cyclophosphamide-Based Chemotherapy

Breast Care ◽

10.1159/000514891 ◽

2021 ◽

pp. 1-7

Author(s):

Jörg Schilling ◽

Christian M. Kurbacher ◽

Claus Hanusch ◽

Steffi Busch ◽

Martin Holländer ◽

...

Keyword(s):

Breast Cancer ◽

Quality Of Life ◽

Real World ◽

Fixed Combination ◽

Complete Response ◽

Nausea And Vomiting ◽

Interventional Study ◽

Beneficial Effects ◽

Real World Evidence

Introduction: In a prospective non-interventional study involving 2,173 patients, we showed that use of the oral fixed combination of netupitant 300 mg and palonosetron 0.5 mg (NEPA) for prevention of chemotherapy (Ctx)-induced nausea and vomiting has beneficial effects on the quality of life (QoL) of patients with various types of cancers receiving highly or moderately emetogenic Ctx. Here, we report on the effects on QoL, effectiveness, and tolerability of NEPA in patients with breast cancer exposed to anthracycline-cyclophosphamide (AC)-based Ctx. Methods: This is a post hoc subanalysis of a prospective non-interventional study in 1,197 patients with breast cancer receiving up to 3 cycles of doxorubicin or epirubicin plus cyclophosphamide and NEPA. NEPA administration was per the summary of product characteristics. Results: In cycle 1 of Ctx, a large proportion of patients (84%) reported “no impact on daily life” (NIDL) due to vomiting; 53% of patients reported NIDL due to nausea. The complete response rate was 86/88/81% in the acute/delayed/overall phase in cycle 1, and NEPA was well tolerated throughout the study. Conclusion: The real-world beneficial effects of NEPA prophylaxis on QoL were confirmed for patients with breast cancer receiving AC. NEPA was effective with a good safety profile in this patient population in clinical practice.

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