Descriptive and Prognostic Value of Patient-Reported Outcomes: The Bortezomib Experience in Relapsed and Refractory Multiple Myeloma

2006 ◽  
Vol 24 (6) ◽  
pp. 976-982 ◽  
Author(s):  
Dominique Dubois ◽  
Ravinder Dhawan ◽  
Helgi van de Velde ◽  
Dixie Esseltine ◽  
Sanjay Gupta ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Survival Analysis ◽  
Functional Assessment ◽  
Patient Reported Outcomes ◽  
Gynecologic Oncology Group ◽  
Life Questionnaire ◽  
Minor Response ◽  
Patient Reported ◽  
The Impact ◽  
Complementary Value

Purpose Bortezomib, a boronic acid dipeptide, has been recently introduced as a new approach to treating multiple myeloma (MM). The goal of this work was to evaluate the added value of patient-reported outcomes (PRO) in the interpretation of bortezomib clinical trial outcomes. Patients and Methods Two hundred two patients with relapsed, refractory MM were treated with bortezomib as part of the SUMMIT (Study of Uncontrolled Multiple Myeloma Managed with Proteasome Inhibition Therapy) study. Patients were administered the following four PRO measures at several time points: the European Organisation for Research and Treatment of Cancer (EORTC) core Quality of Life Questionnaire (QLQ-C30) and the myeloma-specific module (QLQ-MY24), the Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue scale, and the Functional Assessment of Cancer Therapy (FACT)/Gynecologic Oncology Group (GOG) Neurotoxicity (Ntx) scale. Minimal important difference (MID) thresholds were used to define patients as improved, stable, or worsened. A survival analysis was conducted to assess the predictive power of PRO data. Results For the total population, there was a positive change between baseline and best end point. Consistent with the clinical responses, change in PRO scores showed statistically significant differences between response groups with PRO improvement in patients with complete response (CR) or partial response (PR), mostly stable scores in patients with minor response or no change, and deterioration in most scores for patients with progressive disease. Change in scores for neuropathy-related symptoms was reasonably stable. In contrast, fatigue scores significantly improved for patients with CR or PR. When various MID thresholds were applied, the proportion of improved patients exceeded 35% for several domains within all change group definitions. Moreover, survival analysis results demonstrated the additional prognostic information PRO data can provide to supplement clinical data. Conclusion This study demonstrated the complementary value for PRO assessments in further interpreting clinical response, the impact of adverse effects, and patient prognosis in clinical trials.

Changes in patient-reported outcomes in patients diagnosed with and treated for multiple myeloma in the Connect MM registry.

2013 ◽  
Vol 31 (15_suppl) ◽  
pp. 8586-8586 ◽  
Author(s):  
Chris L. Pashos ◽  
Jatin J. Shah ◽  
Howard R. Terebelo ◽  
Brian G. Durie ◽  
Rafat Abonour ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Patient Reported Outcomes ◽  
Performance Status ◽  
Eastern Cooperative Oncology Group ◽  
Staging System ◽  
Ecog Performance Status ◽  
Related Factors ◽  
Patient Reported ◽  
Ecog Ps ◽  
The Impact

8586 Background: Little is known about the impact of treatment on patient-reported outcomes (PROs) and health-related quality of life (HRQoL) in multiple myeloma (MM) patients (pts). The change in PROs of MM pts between baseline and 1 year was assessed relative to their baseline International Staging System (ISS) stage and Eastern Cooperative Oncology Group (ECOG) performance status (PS) score. Methods: Connect MM is a prospective US registry of MM pts initiated in 2009. Clinicians reported pt demographics, ECOG PS score, and ISS stage. PROs were collected at baseline and at 1 year utilizing the Functional Assessment of Cancer Therapy (FACT)-MM, EQ-5D, and Brief Pain Inventory (BPI). Changes in FACT-MM, EQ-5D, and BPI scores were analyzed by ISS stage and ECOG PS score in 636 pts meeting CRAB criteria from 189 centers. Results: Most pts were male (58%) and white (84%). Mean age was 66 years (± 11). Pts were treated in community (81%), academic (17%), or veterans/military (2%) settings. ISS stages of pts were: I (29%), II (35%), and III (35%). ECOG PS scores were 0 (37%), 1 (49%), 2 (11%), and 3 (3%). Improvements in overall HRQoL as shown by the FACT-MM and FACT-General (G) total scores, were observed across all ISS stages (P = 0.03 to < 0.0001) with no significant differences between stages. Improvements in FACT-MM and FACT-G total scores were observed with ECOG PS scores 1–3 (P = 0.03 to 0.005). Pts with poorer ECOG PS scores tended to have greater improvement in EQ-5D domains of mobility, self-care, and usual activities. HRQoL/functional ability improved in 4 of 5 FACT domains (except social/family; all others P < 0.0001), and in 4 of 5 EQ-5D domains (except pain/discomfort; all others, P = 0.01 to < 0.0001). BPI showed that overall average pain improved (P < 0.0005) over 1 year, but statistically significant differences by ISS stage or ECOG PS score were not observed. Conclusions: Connect MM data showed that overall HRQoL of MM pts improved between baseline and 1 year, with a consistent benefit observed across pts with different ISS stages and ECOG PS scores. Additional analysis should examine which disease- and treatment-related factors are associated with these HRQoL improvements.

The Prognostic Significance of Patient-Reported Outcomes in Cancer Clinical Trials

2008 ◽  
Vol 26 (8) ◽  
pp. 1355-1363 ◽  
Author(s):  
Carolyn C. Gotay ◽  
Crissy T. Kawamoto ◽  
Andrew Bottomley ◽  
Fabio Efficace
Keyword(s):  
Quality Of Life ◽  
Clinical Trials ◽  
Patient Reported Outcomes ◽  
Prognostic Significance ◽  
Cancer Clinical Trials ◽  
Life Questionnaire ◽  
European Organization ◽  
Patient Reported ◽  
The Impact

Purpose Patient-reported outcomes (PROs), routinely collected as a part of cancer clinical trials, have been linked with survival in numerous clinical studies, but a comprehensive critical review has not been reported. This study systematically assessed the impact of PROs on patient survival after a cancer diagnosis within the context of clinical trials. Design Cancer clinical trials that assessed baseline PROs and mortality were identified through MEDLINE (through December 2006) supplemented by the Cochrane database, American Society of Clinical Oncology/European Society for Medical Oncology abstracts and hand searches. Inclusion criteria were publication in English language and use of multivariate analyses of PROs that controlled for one or more clinical factors. Two raters reviewed each study, abstracted data, and assessed study quality; two additional raters verified abstractions. Results In 36 of 39 studies (N = 13,874), at least one PRO was significantly associated with survival (P < .05) in multivariate analysis, with varying effect sizes. Studies of lung (n = 12) and breast cancer (n = 8) were most prevalent. The most commonly assessed PRO was quality of life, measured by the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire C30 in 56% of studies. Clinical variables adjusted for included performance status (PS), treatment arm, stage, weight loss, and serum markers. Results indicated that PROs provide distinct prognostic information beyond standard clinical measures in cancer clinical trials. Conclusion PROs might be considered for stratification purposes in future trials, as they were often better predictors of survival than PS. Studies are needed to determine whether interventions that improve PROs also increase survival and to identify explanatory mechanisms through which PROs relate to survival.

Financial toxicity in patients with multiple myeloma participating in clinical trials: A U.S. Food and Drug Administration pooled analysis.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. e19370-e19370
Author(s):  
Bellinda King-Kallimanis ◽  
Ting-Yu Chen ◽  
Bindu Kanapuru ◽  
Vishal Bhatnagar ◽  
Paul Gustav Kluetz
Keyword(s):  
Multiple Myeloma ◽  
Clinical Trials ◽  
Medical Treatment ◽  
Physical Condition ◽  
Economic Status ◽  
Pooled Analysis ◽  
Life Questionnaire ◽  
Financial Toxicity ◽  
Patient Reported ◽  
The Impact

e19370 Background: Financial toxicity (FT) is a major concern for patients receiving standard cancer treatment, and FT can lead to worse cancer outcomes. However, little is known about the FT of patients enrolled in clinical trials (CT). While investigational treatment may be provided by sponsors free of charge, patients are subject to increased clinic visits (ie, missed workdays, travel costs) and still bear the cost of usual care. This analysis evaluates patient-reported FT in multiple myeloma (MM) trials submitted to US FDA and explores the relationship between baseline FT and overall response rate (ORR). Methods: We pooled data from 9 MM registration CTs submitted to the FDA that included the EORTC Quality of Life questionnaire (QLQ-C30). The QLQ-C30 includes an item asking patients “Has your physical condition or medical treatment caused you financial difficulties?”. We looked at proportion of patients at baseline reporting any FT and their ORR. We also report prevalence, incidence and change from baseline FT at 3 and 6 months. Results: 5,667 patients answered the FT item. Mean age of patients was 65 years, 55% were male, 85% were white and 6% were enrolled in the US. Approximately a third of patients reported experiencing any FT at baseline, 3 and 6 months. When compared to their baseline, 69% of patients had unchanged FT and 14% had worse FT at both 3 and 6 months. The incidence of new cases of FT at 3 months was 17% and at 6 months 7%. The ORR in patients who reported any FT at baseline was 69% compared to 72% in patients with no baseline FT, and was similar regardless of the degree of FT. Conclusions: Few studies have investigated FT in a large sample of CT participants. In this pooled analysis of patients enrolled in MM CTs, approximately a third of patients reported FT associated with their physical condition or medical treatment at baseline and throughout the trial. A small percentage of patients experienced worsening FT during the trial and a small percentage went from no FT to reporting any FT. The impact of baseline FT on ORR was small. Limitations of this preliminary analysis include missing patient level or group level insurance status, socio-economic status and other important determinants of health.

Patient-Reported Outcomes After Hepatic Resection of Colorectal Cancer Metastases

2012 ◽  
Vol 30 (12) ◽  
pp. 1364-1370 ◽  
Author(s):  
Jonathan R. Rees ◽  
Jane M. Blazeby ◽  
Peter Fayers ◽  
Elizabeth A. Friend ◽  
Fenella K.S. Welsh ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Liver Metastases ◽  
Hepatic Resection ◽  
Patient Reported Outcomes ◽  
Hepatic Metastases ◽  
Life Questionnaire ◽  
Quality Of Life Questionnaire ◽  
Patient Reported ◽  
The Impact

Purpose Hepatic resection of colorectal carcinoma (CRC) liver metastases is increasing, but evidence for the impact of surgery on patient-reported outcomes (PROs) is limited. This study aimed to describe comprehensively the impact of liver surgery for CRC hepatic metastases on PROs. Patients and Methods Consecutive patients selected for hepatic resection completed the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire–C30 and Quality of Life Questionnaire–Liver Metastases C21 before and 3, 6, and 12 months after surgery. For functional scales, mean scores with 95% CIs were calculated at each time point, with differences in scores of at least 10 points considered clinically significant. Responses to symptom scales and items were categorized as minimal or severe. Proportions and 95% CIs for each symptom category were calculated. Results Hepatic surgery was planned in 241 patients but abandoned in nine because of unresectable disease. There were two postoperative deaths, 58 complications (25.2%), and 32 patients (14.9%) with disease recurrence. Questionnaire compliance was excellent (> 95% at all time points). After surgery, most functional aspects of health decreased, and the proportions of patients with severe symptoms increased; role function deteriorated significantly, and 30% of patients reported severe activity/vigor problems. Functional scales recovered by 6 months and were maintained at 1 year. Postoperative symptoms returned to baseline levels at 12 months, but 32.1% of patients reported severe problems with sexual dysfunction and 11.9% with abdominal pain. Conclusion These findings provide new evidence regarding outcomes of liver resection for CRC metastases. It is recommended that patients be reassured that surgery has a minimal and short-lived detrimental impact on health.

Patient-reported outcomes in clinical trials for multiple myeloma: Where we are.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e18615-e18615
Author(s):  
Marike Andreas ◽  
Sam Salek ◽  
Nina Kreuzberger ◽  
Vanessa Piechotta ◽  
Esther Natalie Oliva ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Randomized Controlled Trials ◽  
Study Protocol ◽  
Patient Reported Outcomes ◽  
Controlled Trials ◽  
Trial Registry ◽  
Randomized Controlled ◽  
Patient Reported ◽  
The Impact ◽  
Pro Instruments

e18615 Background: It is recommended to include patient-reported outcomes (PROs) in cancer trials to ensure clinical benefit based on patients’ perceptions. For multiple myeloma patients, PRO measurement is essential to understand the impact of treatment on their physical and psychosocial functional behaviour. However, it is unclear which PRO instruments are utilized in multiple myeloma trials, how often they are used and whether results are consistently reported. The aim of this project was to explore the use and frequency of PRO instruments in randomized controlled trials for multiple myeloma and to assess the consistency of their reporting. Methods: Within the European Hematology Association (EHA) project to develop guidelines for the use of PROs in adult patients with hematological malignancies, MEDLINE and CENTRAL were systematically searched for randomized controlled trials investigating multiple myeloma between 2015 and 2020. Study design, disease and treatment characteristics, the primary outcome and used PRO instrument(s) were extracted using a pre-defined template. To assess the consistency of PRO reporting, study registries were compared with publications, study protocol and Health Technology Assessment (HTA) reports where available. Results: Overall, 10,707 records were found. Following screening for randomized-controlled trials, 283 ongoing, completed or published studies were included for review. 118 studies planned the use of PROs. The most frequently used PRO instrument (92 studies) was the EORTC QLQ-30, an instrument that measures health-related quality-of-life (HRQoL) in cancer patients. The disease-specific questionnaire EORTC-MY20 was also frequently used (50 studies). Likewise, the HRQoL instrument EQ-5D was used in 50 studies. Overall, 38 different PRO instruments were reported. In 39 studies for which a study protocol was found, only 19 reported PRO instruments consistently with the trial registry for the study. In addition, in 30 studies, for which a publication reported PRO results (58 overall), the information on PRO instruments differed between publication and the trial registry. For example, PRO instruments mentioned in the registry were omitted in the respective publications. Additionally, information on PRO in HTA reports was available for 26 studies, of which 18 reports were consistent with the trial registries. Conclusions: The results show that measurement of PROs still remain under-utilized in multiple myeloma research. While most PRO instruments identified in this review comply with the recommendations for incorporating PROs into clinical research in adult oncology, they are often inconsistently reported. Thus, reporting standards for the use of PROs are needed to ensure consistency in the use and reporting of PROs.

scholarly journals Patient‐reported outcomes following autologous stem cell transplant for patients with multiple myeloma

eJHaem ◽  
2021 ◽  
Author(s):  
Noa Biran ◽  
Wanting Zhai ◽  
Roxanne E. Jensen ◽  
Jeanne Mandelblatt ◽  
Susan Kumka ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Stem Cell ◽  
Patient Reported Outcomes ◽  
Stem Cell Transplant ◽  
Autologous Stem Cell Transplant ◽  
Cell Transplant ◽  
Patient Reported

Overview of the Patient Perspective at OMERACT 10 — Conceptualizing Methods for Developing Patient-Reported Outcomes

2011 ◽  
Vol 38 (8) ◽  
pp. 1699-1701 ◽  
Author(s):  
JOHN R. KIRWAN ◽  
PETER S. TUGWELL
Keyword(s):  
Clinical Trials ◽  
Experimental Work ◽  
Patient Reported Outcomes ◽  
Patient Perspective ◽  
Methodological Issues ◽  
Instrument Selection ◽  
Patient Reported ◽  
The Impact ◽  
Choice Of Instruments ◽  
The Way

This overview draws out the main conclusions from the 4 workshops focused on incorporating the patient perspective into outcome assessment at the 10th Outcome Measures in Rheumatology (OMERACT 10) conference. They raised methodological issues about the choice of outcome domains to include in clinical trials, the development or choice of instruments to measure these domains, and the way these instruments might capture the impact of a disease and its treatment. The need to develop a more rigorous conceptual model of quantifying the way conditions affect health, and the need to ensure patients are directly involved in the decisions about domains and instruments, emerged clearly. The OMERACT participants voted to develop guidelines for domain and instrument selection, and conceptual and experimental work will be brought forward to revise and upgrade the OMERACT Filter.

The impact of a recent relapse on patient-reported outcomes in subjects with multiple sclerosis

2012 ◽  
Vol 21 (10) ◽  
pp. 1677-1684 ◽  
Author(s):  
Brian C. Healy ◽  
Irene R. Degano ◽  
Ana Schreck ◽  
David Rintell ◽  
Howard Weiner ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Patient Reported Outcomes ◽  
Patient Reported ◽  
The Impact

Patient reported outcomes in patients with desmoid type fibromatosis.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 11574-11574
Author(s):  
VIKAS GARG ◽  
Sameer Rastogi ◽  
Adarsh Barwad ◽  
Rambha Panday ◽  
Sandeep Kumar Bhoriwal ◽  
...  
Keyword(s):  
Patient Reported Outcomes ◽  
Symptom Burden ◽  
Benign Neoplasm ◽  
Anxiety And Depression ◽  
Appendicular Skeleton ◽  
Healthy Controls ◽  
Symptom Scale ◽  
Cross Sectional ◽  
Patient Reported ◽  
The Impact

11574 Background: Desmoid type fibromatosis (DTF) is a rare benign neoplasm with infiltrative growth and high local recurrences. Due to long disease course, unpredictable growth pattern, and low mortality, using only survival outcomes may be inappropriate. In this study we assessed the impact of DTF on health related quality of life (HRQoL). Methods: This was a cross-sectional study done in patients with DTF. The study participants were asked to fill the EORTC QLQ-C30, GAD-7 and PHQ- 9 q uestionnaires to assess HRQoL, anxiety and depression . Outcomes were also compared with healthy controls. Results: 204 subjects (102 DTF patients and 102 healthy controls) were recruited. Study parameters have been summarized in Table. Appendicular skeleton (limbs + girdle) was most commonly involved in 59 % patients and abdominal wall or mesentery was involved in 22.5 %. Patients have received median of 2 lines of therapy. 54 % patients were currently on sorafenib and 41 % were under active surveillance. Mean global health status in DTF patient 65.58 ± 22.64, was significantly lower than healthy controls. Similarly, DTF patients scored low on all functional scales except cognitive functioning. Symptom scale showed significantly higher symptom burden of fatigue, pain, insomnia and financial difficulties. Anxiety & depression was observed in 39.22 % and 50 % of DTF patients respectively. DTF patients had higher rates of mild, moderate and severe anxiety and depression compared to healthy controls. No difference was observed based on site of disease. Conclusions: DTF patients have significant symptom burden, poor functioning, and heightened anxiety and depression. Patient reported outcomes should be routinely used to assess treatment efficacy in DTF patients.[Table: see text]

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