scholarly journals The Associations between Coping Strategy Use and Patient-Reported Outcomes in Patients with Acute Myeloid Leukemia

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 4131-4131
Author(s):  
Matthew J. Reynolds ◽  
Monica Bodd ◽  
Ashley Nelson ◽  
Richard Newcomb ◽  
P. Connor Johnson ◽  
...  
Keyword(s):  
Psychological Distress ◽  
Coping Strategies ◽  
Coping Strategy ◽  
Research Funding ◽  
Patient Reported Outcomes ◽  
Advisory Board ◽  
Strategy Use ◽  
Avoidant Coping ◽  
Patient Reported ◽  
High Utilization

Abstract Background: Patients with acute myeloid leukemia (AML) who receive intensive chemotherapy must cope with immense physical and psychological symptoms associated with a variety of patient-reported outcomes (PROs) such as quality of life (QOL). Although coping is critical to the management of an AML diagnosis and its treatment, data characterizing the use of coping strategies and its associations with PROs in the AML population are limited. Hence, we characterize coping strategy use among patients with AML and examine the associations between coping strategy use, psychological distress, and QOL. Methods: We used cross-sectional secondary data analyses to describe coping in 160 patients with newly diagnosed high-risk AML enrolled in a multi-site randomized supportive care trial. We used the Brief COPE, Hospital Anxiety and Depression Scale (HADS), PTSD Checklist-Civilian Version (PCL-C), and Functional Assessment of Cancer Therapy-Leukemia (FACT-Leu) within 72 hours of patient initiation of chemotherapy, to measure coping strategies, psychological distress and QOL, respectively. We grouped coping strategies into two higher-order domains of coping based on prior literature: approach-oriented coping (i.e., use of emotional support, active coping, positive reframing, acceptance) or avoidant coping (i.e., self-blame, denial, behavioral disengagement). We used the median split method for the distribution of coping domains. We used multivariate regression models adjusting for age, gender and diagnosis type (newly diagnosed vs. relapsed/refractory AML) to assess the relationship between coping and PROs. Results: Participants (median age of 64.4 years) were mostly non-Hispanic White (86.3%), male (60.0%), and married (73.8%). Most (51.9%) reported high utilization of approach-oriented coping strategies (e.g., emotional support) whereas 38.8% reported high utilization of avoidant coping strategies (e.g., denial) (Figure 1). At the time of AML diagnosis, use of approach-oriented coping was associated with less psychological distress and better QOL (Table 1). Use of avoidant coping was associated with more psychological distress and worse QOL. Additionally, patients who used multiple approach-oriented coping strategies had less psychological distress and better QOL (Table 2). In contrast, patients who used multiple avoidant coping strategies had more psychological distress, and worse QOL. Conclusions: Our study illustrates that most patients with high-risk AML utilize both approach-oriented and avoidant coping strategies. Our results also reveal links between approach-oriented coping strategies, less psychological distress, and better QOL. These findings underscore the need for early integration of supportive oncology interventions that help patients to cultivate approach-oriented coping strategies. Figure 1 Figure 1. Disclosures Brunner: Novartis: Consultancy, Research Funding; BMS/Celgene: Consultancy, Research Funding; Takeda: Consultancy, Research Funding; Acceleron: Consultancy; Agios: Consultancy; Keros Therapeutics: Consultancy; GSK: Research Funding; Aprea: Research Funding; AstraZeneca: Research Funding; Janssen: Research Funding. Fathi: AbbVie: Consultancy, Honoraria, Research Funding; Takeda: Consultancy, Honoraria; Pfizer: Consultancy, Honoraria; Blueprint: Consultancy, Honoraria; Seattle Genetics: Consultancy, Honoraria; Astellas: Consultancy, Honoraria; Daiichi Sankyo: Consultancy, Honoraria; Genentech: Consultancy, Honoraria; Trillium: Consultancy, Honoraria; Kura: Consultancy, Honoraria; Foghorn: Consultancy, Honoraria; Kite: Consultancy, Honoraria; Morphosys: Consultancy, Honoraria; Ipsen: Consultancy, Honoraria; Agios: Consultancy, Honoraria, Research Funding; Servier: Research Funding; Celgene/BMS: Consultancy, Honoraria, Research Funding. LeBlanc: Seattle Genetics: Consultancy, Other: Advisory board, Research Funding; Pfizer: Consultancy, Other: Advisory Board; AstraZeneca: Consultancy, Honoraria, Other: Advisory board, Research Funding; UpToDate: Patents & Royalties; American Cancer Society: Research Funding; Agios: Consultancy, Honoraria, Other: Advisory board; Travel fees, Speakers Bureau; BMS/Celgene: Consultancy, Honoraria, Other: Travel fees, Research Funding, Speakers Bureau; Daiichi-Sankyo: Consultancy, Honoraria, Other: Advisory board; Flatiron: Consultancy, Other: Advisory board; Astellas: Consultancy, Honoraria, Other: Advisory board; AbbVie: Consultancy, Honoraria, Other: Advisory board; Travel fees, Speakers Bureau; Otsuka: Consultancy, Honoraria, Other; Jazz Pharmaceuticals: Research Funding; Duke University: Research Funding; Helsinn: Consultancy, Research Funding; Heron: Consultancy, Honoraria, Other: advisory board; CareVive: Consultancy, Other, Research Funding; NINR/NIH: Research Funding; Amgen: Consultancy, Other: travel.

scholarly journals Coping Strategies in Patients with Acute Myeloid Leukemia

2021 ◽  
Author(s):  
Hermioni L. Amonoo ◽  
Monica H. Bodd ◽  
Matthew J. Reynolds ◽  
Ashley M. Nelson ◽  
Richard A. Newcomb ◽  
...  
Keyword(s):  
Psychological Distress ◽  
Coping Strategies ◽  
Myeloid Leukemia ◽  
Patient Reported Outcomes ◽  
Avoidant Coping ◽  
Depression Symptoms ◽  
Ptsd Symptoms ◽  
Patient Reported ◽  
High Utilization ◽  
Acute Myeloid

Patients diagnosed with acute myeloid leukemia (AML) face sudden-onset life-threatening disease that requires intensive treatments. Although their early disease trajectory is characterized by significant, toxic side effects, there is limited data describing coping strategies among patients with AML and how these inform patient-reported outcomes. We used cross-sectional secondary data analyses to describe coping in 160 patients with newly diagnosed high-risk AML. We used the Brief COPE, Hospital Anxiety and Depression Scale, PTSD Checklist-Civilian Version, and Functional Assessment of Cancer Therapy-Leukemia at time of AML diagnosis to measure coping strategies, psychological distress and quality of life (QOL), respectively. We used the median split method for distribution of coping domains, and multivariate regression models to assess the relationship between coping and patient-reported outcomes. Participants (median age=64.4 years) were mostly non-Hispanic White (86.3%), male (60.0%), and married (73.8%). Most (51.9%) had high utilization of approach-oriented coping strategies whereas 38.8% had high utilization of avoidant coping strategies. At time of diagnosis, use of approach-oriented coping was associated with less psychological distress (anxiety: β=-0.262, p=0.002; depression symptoms β=-0.311, p<0.001; PTSD symptoms: β=-0.596, p=0.006) and better QOL (β=1.491, p=0.003). Use of avoidant coping was associated with more psychological distress (anxiety: β=0.884, p<0.001; depression symptoms: β=0.697, p<0.001; PTSD symptoms: β=3.048, p<0.001) and worse QOL (β=-5.696, p<0.001). Patients with high-risk AML utilize various approach-oriented and avoidant coping strategies at time of diagnosis. Use of approach-oriented coping strategies was associated with less psychological distress and better QOL, suggesting a possible target for supportive oncology interventions.

scholarly journals Patient Reported Outcomes (PROs) Show Superior Quality of Life (QOL) of Patients with Chronic Myeloid Leukemia (CML) Treated with First Compared to Newer Generations of Tyrosine Kinase Inhibitors (TKIs)

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 4781-4781
Author(s):  
Adi Shacham ◽  
Lena Rosenman ◽  
Avi Leader ◽  
Giora Sharf ◽  
Pia Raanani ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Body Image ◽  
Myeloid Leukemia ◽  
Research Funding ◽  
Patient Reported Outcomes ◽  
Kinase Inhibitors ◽  
Advisory Board ◽  
Daily Activities ◽  
Patient Reported

Background: Tyrosine kinase inhibitors (TKIs) prolong the survival of patients with chronic myeloid leukemia (CML). Nowadays, the life expectancy of these patients is approaching that of an age-matched population. Since imatinib, was first introduced 20 years ago, several newer more potent TKIs, have been approved for CML. While these drugs are effective, they come with the trade-off of adverse effects, some are common to all and some are drug-specific. Most patients will receive prolonged treatment, therefore quality of life (QOL) is becoming a major concern. QOL under these drugs has not been directly compared. Herein, we compared the QOL of patients with CML treated with imatinib, dasatinib and nilotinib using patient reported outcomes (PROs) questionnaire. Methods: This nationwide study was a joint initiative of the Israeli CML patients' organization and the division of hematology at Rabin Medical Center, Israel. Patients completed computerized questionnaires that were provided to us by the European Organization for Research and Treatment of Cancer (EORTC). The questionnaires included 30 items core questionnaire (QLQ-C30), 24 items of CML-specific questionnaire (QLQ-CML24) and additional items that were added by the researchers. Questioners are composed of functional, symptom and global health/QOL scales/items. All scales and single-item measures were standardized and the score ranges from 0 to 100. High score for functional and QOL items/scales represents better function and QOL. High score in symptoms items represents worse symptomatology. We used the Mann-Whitney test to compare medians and χ2 to compare categorical variables. Results: Overall, 195 patients completed the questionnaire. The median age was 58 years (range: 23 to 89) and of those 102 patients (59%) were males. Time from diagnosis ranged between less than a year and 46 years (median: 7 years). In the primary analysis we included 139 patients (71%) who received either imatinib (n = 70, 36%), dastainib (n = 45, 23%) or nilotinib (n = 24, 12%). We did not include the few patients who received bosutinib (n = 8, 4%), ponatinib (n = 2, 1%) or patients who discontinued treatment (n = 22, 11%). Patients on imatinib were older (median age 67 years, range : 32 to 89) compared with patients on either nilotinib (median age 50 years, range 26 to 85 ) or dasatinib (median age 47 years, range: 26 to 85) (P<0.0001), but had similar demographics and disease characteristics. The EORTC questionnaires included 34 items related to symptoms experienced by patients within the last week. These items were grouped into 13 symptom scales. Overall, the symptomatic profile was more severe in patients who received either dasatinib or nilotinib compared with imatinib. For example, compared with patients who received dasitinib or nilotinib, patients on imatinib experienced lesser limitation on daily activities (38 and 47 vs. 25 respectively, P = 0.02), less fatigue (53 and 57 vs. 37 respectively, P = 0.001) and lesser degree of impaired body image (38 and 46 vs. 26 respectively, P = 0.022). Likewise, symptom burden score was 41 in patients on nilotinib, higher than in patients receiving dasatinib (27, P = 0.005) or imatinib (30, P = 0.018). In addition, patients on imatinib had less painful episodes compared with patients on nilotinib (28 vs. 47. P = 0.014). The questionnaire included also 18 items that were grouped into 8 scales which estimated their functional status. Compared with nilotinib, patients on imatinib had better emotional functioning (77 vs. 61, P = 0.009); patients were less worried, stressed depressed or nervous. Patients were also more satisfied of the knowledge and treatment they received by their caregivers (41 vs. 19 24, P = 0.01). Finally, although patients on imatinib were older, they reported better home and work function (67) compared with either nilotinib (50) or dasatinib (50) (P < 0.001). Conclusions: Compared with nilotinib and dasatinib, patients who received imatinib reported lower levels of symptomatology. They reported less fatigue, limitation of daily activities, impaired body image and pain. They were also more satisfied by their caregiver, had better emotional functioning and were less worried, depressed or nervous. Since patients on 2nd generation TKIs were almost 2 decades younger, these differences reflect not only the TKI toxicity profile but also the generation-gap and the primary significance "Generation Y" gives to QOL issues. Disclosures Shacham: novartis: Consultancy. Sharf:Janssen: Other: Advocacy grants funding; Novartis: Honoraria, Other: Advocacy Advisory Board, Research Funding; Abbvie: Other: Advocacy grants funding; Takeda: Other: Advocacy grants funding; Incyte: Honoraria, Other: Advocacy Advisory Board, Research Funding; Pfizer: Honoraria, Other: Advocacy Advisory Board, Research Funding; BMS: Other: Advocacy grants funding, Research Funding; Roche: Other: Advocacy grants funding.

Coping strategies, trajectories, and their associations with patient-reported outcomes among women with ovarian cancer

2018 ◽  
Vol 26 (12) ◽  
pp. 4133-4142 ◽  
Author(s):  
Vanessa L. Beesley ◽  
◽  
David D. Smith ◽  
Christina M. Nagle ◽  
Michael Friedlander ◽  
...  
Keyword(s):  
Ovarian Cancer ◽  
Coping Strategies ◽  
Patient Reported Outcomes ◽  
Patient Reported

scholarly journals Role of Patient Coping Strategies in Understanding the Effects of Early Palliative Care on Quality of Life and Mood

2018 ◽  
Vol 36 (1) ◽  
pp. 53-60 ◽  
Author(s):  
Joseph A. Greer ◽  
Jamie M. Jacobs ◽  
Areej El-Jawahri ◽  
Ryan D. Nipp ◽  
Emily R. Gallagher ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Palliative Care ◽  
Depressive Symptoms ◽  
Coping Strategies ◽  
Indirect Effect ◽  
Avoidant Coping ◽  
Intervention Effects ◽  
Early Palliative Care ◽  
Patient Reported

Purpose The early integration of oncology and palliative care (EIPC) improves quality of life (QOL) and mood for patients with advanced cancer. However, the mechanisms by which EIPC benefits these outcomes remain unclear. We therefore examined whether EIPC improved patients’ coping strategies and if changes in coping accounted for intervention effects on QOL and depressive symptoms. Patients and Methods For this secondary analysis of an EIPC trial, we examined data from 350 patients with newly diagnosed incurable lung or GI cancer. Participants completed assessments of QOL (Functional Assessment of Cancer Therapy–General), depressive symptoms (Patient Health Questionnaire–9), and coping (Brief COPE) at baseline and 24 weeks. We used linear regression to test intervention effects on use of coping strategies and mediation regression models with bias-corrected bootstrapping to examine whether improvements in coping mediated the effects of early palliative care on patient-reported outcomes. Results Compared with usual oncology care, EIPC significantly increased patient use of approach-oriented coping strategies ( B = 1.09; SE = 0.44; P = .01) and slightly reduced use of avoidant strategies ( B = −0.44; SE = 0.23; P = .06) from baseline to 24 weeks. Also, the increased use of approach-oriented coping and reduction in avoidant coping were associated with higher QOL and lower depressive symptoms at 24 weeks. The positive changes in approach-oriented coping, but not avoidant coping, significantly mediated the effects of EIPC on QOL (indirect effect, 1.27; 95% CI, 0.33 to 2.86) and depressive symptoms (indirect effect, −0.39; 95% CI, −0.87 to −0.08). Conclusion Patients with incurable cancer who received EIPC showed increased use of approach-oriented coping, which was associated with higher QOL and reduced depressive symptoms. Palliative care may improve these outcomes by providing patients with the skills to cope effectively with life-threatening illness.

Toxic leadership: emotional distress and coping strategy

2019 ◽  
Vol 22 (1) ◽  
pp. 65-78 ◽  
Author(s):  
Asha Bhandarker ◽  
Snigdha Rai
Keyword(s):  
Psychological Distress ◽  
Coping Strategies ◽  
Coping Strategy ◽  
Adaptive Coping ◽  
Toxic Leadership ◽  
Avoidance Coping ◽  
Content Type ◽  
Loss Of Self ◽  
Self Worth ◽  
And Coping

Purpose The purpose of this paper is to elucidate the distressing impact of toxic leadership on the mental state of the subordinates and examine the unique coping mechanisms used by them to deal with such leaders. The paper also examined the relationship between psychological distress and coping strategy used by subordinates to deal with the toxic leader. Design/methodology/approach This study presents a validity testing of two scales. The first scale was designed to measure experienced psychological distress emanating from exposure to toxic leaders, and the second scale aims to assess the coping strategies utilized by subordinates to deal with the toxic leaders. Data were collected from 570 employees working in public as well as private organizations in India. Findings The results of this paper supported the theorized two three-dimensional tools to measure: psychological distress (loss of self-worth, withdrawal and agitated) and coping strategies to deal with toxic leaders (assertive coping, avoidance coping and adaptive coping). Reliability estimates and construct validity of both the tools were established. The results also suggest that the loss of self-worth was negatively related with assertive coping, avoidance coping and adaptive coping. However, withdrawal was positively related with assertive coping and avoidance coping. Finally, agitation was positively related with avoidance and adaptive coping. Originality/value To the authors’ knowledge, this is one of the rare studies to examine together the phenomenon of both psychological distress experienced by subordinates and the coping strategies utilized by them to deal with toxic leaders.

Prospective Validation of the Italian Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF: Italian) In 186 MPN Patients

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 5060-5060
Author(s):  
Robyn Scherber ◽  
Tiziano Barbui ◽  
Alessandro M. Vannucchi ◽  
Francesco Passamonti ◽  
Giovanni Barosi ◽  
...  
Keyword(s):  
Research Funding ◽  
Patient Reported Outcomes ◽  
Myeloproliferative Neoplasm ◽  
Office Visit ◽  
Symptom Assessment ◽  
Advisory Committees ◽  
Patient Reported ◽  
Eortc Qlq C30 ◽  
The Usa ◽  
Eortc Qlq

Abstract Abstract 5060 Background: The Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) is a concise instrument of patient reported outcomes (PRO) designed to assess the unique spectrum of symptoms present in the majority of patients (Mesa et. al. Cancer 2007). We sought to validate the Italian Translation of the MPN-SAF which addresses 19 separate symptoms reported by MPN patients. Methods: Translation: We utilized the standard practice of PRO translation in which 3 independent translations are created by 3 independent translators fluent in both languages. A fourth translator then discussed the translations with the other translators and a consensus translation was obtained. Validation: Patients self completed the MPN-SAF: Italian at the time of a physician office visit and the Italian EORTC-QLQ-C30 (a widely used instrument of PRO for cancer patients) was co-administered for validation purposes. Results: Patients and Symptomatic Burden: 186 patients were prospectively enrolled (ET (N=88; 47%), PV (N=69; 37%) and MF (N=29; 16%)) a median of 6 years (range:0-29) from their diagnosis. Patients were of a median age (62; range 29–91 years) and gender (56% females) typical of the disease. 72% (N=135) had received some form of non-aspirin medical therapy for their disease, and 68% were on therapy at the time of completing the questionnaire. Patients frequently had a history of either thrombotic events (31%) and/or hemorrhagic events (13%). The MPN-SAF measured 19 items in the enrolled patients (data summarized in Table 1). Validation Analysis: EORTC-QLQ-C30: Consistent with our experience with the MPN-SAF:English, Pearson correlations between MPN-SAF:Italian individual symptom scores and the Italian EORTC-QLQ C30 showed excellent correlations with co-validation questions including fatigue, pain, insomnia, (all p<0.001). Excellent correlations were demonstrated between EORTC-QLQ-C30 subscales and corresponding MPN-SAF measurements. Comparison with MPN-SAF:English: Comparison with 102 patients prospectively completing the MPN-SAF: English (ET=20, PV=23, MF=59) in the USA indicated very strong correlations (when controlling for MPN subtype) in the prevalence of all 19 items assessed and only subtle differences in terms of symptomatic severity for fatigue, itching and insomnia. Conclusions: The MPN-SAF:Italian is an easy to administer, clear, 19-item inventory of patient-reported outcomes that is specific to MPNs. Additionally, the instrument is validated by 1) comparison to previously validated Italian instruments and 2) the correlation with the MPN-SAF:English. Utilization of the instrument in Italian MPN clinical trials will allow for useful comparison to patients completing the MPN-SAF in other countries and will serve as a valuable clinical marker of disease symptom severity. Disclosures: Vannucchi: Novartis: Membership on an entity's Board of Directors or advisory committees. Mesa:SBio: Research Funding; Novartis: Research Funding; Celgene: Research Funding; Incyte: Research Funding; Roche: Research Funding; eisai: Research Funding; telik: Research Funding.

The association between physical functioning, symptom burden, and coping strategies with quality of life (QOL) in patients with chronic graft-versus-host disease (cGVHD).

2018 ◽  
Vol 36 (34_suppl) ◽  
pp. 178-178
Author(s):  
Robert Sommer ◽  
Jamie M. Jacobs ◽  
Lauren Waldman ◽  
Lara Traeger ◽  
Joseph Greer ◽  
...  
Keyword(s):  
Psychological Distress ◽  
Coping Strategies ◽  
Physical Functioning ◽  
Depression Scale ◽  
Symptom Burden ◽  
Cell Transplant ◽  
Symptom Scale ◽  
Patient Reported ◽  
And Coping ◽  
Over Time

178 Background: Allogeneic stem cell transplant survivors with cGVHD experience substantial psychological distress and impairments in QOL. However, the relationship between patients’ physical functioning, symptom burden, coping strategies, and QOL over time is unknown. Methods: We conducted a longitudinal study of patients with moderate-severe cGVHD. We assessed patient-reported psychological distress (Hospital Anxiety and Depression Scale), QOL (Functional Assessment of Cancer Therapy-General), physical functioning (Human Activity Profile), cGVHD symptom burden (Lee Symptom Scale), and coping (Coping Inventory for Stressful Situations) at baseline, 3 months, and 6 months. Using mixed linear effects models, we examined the longitudinal relationship between QOL and physical functioning, cGVHD symptoms, and coping strategies. Results: We enrolled 53 patients with moderate (71.7%, 38/53) or severe (28.3%, 15/53) cGVHD. The rate of clinically significant depression and anxiety symptoms at baseline was 32.1% (17/53) and 30.2% (16/33), respectively, and did not change over time. Patients reported low QOL at baseline [M = 70.33, SD = 18.96], which did not change significantly over time [β = -0.66, SE = 1.11, P = 0.550]. Over time, higher physical functioning was associated with better QOL [β = 0.17, SE = 0.05, P = 0.001], while greater symptom burden was associated with worse QOL [β = -0.38, SE = 0.06, P < 0.001]. While the use of emotion-oriented coping was associated with lower QOL over time [β = -0.70, SE = 0.14, P < 0.001], the use of avoidance-oriented coping was associated with higher QOL over time [β = 0.38, SE = 0.10, P < 0.001]. Task-oriented coping was not associated with psychological distress or QOL. Conclusions: Patients with moderate-severe cGVHD report substantial psychological distress and persistently impaired QOL over time. Higher physical function and lower symptom burden are associated with improved QOL. The use of certain coping strategies was associated with changes in QOL. These data underscore the need for supportive care interventions to promote effective coping and enhance physical functioning in patients with cGVHD.

scholarly journals Correlation Between Treatment Outcomes, Baseline Characteristics and Molecular Responses in the Majic Study Which Compared Ruxolitinib to Best Available Therapy in Essential Thrombocythemia

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 1929-1929
Author(s):  
Claire N. Harrison ◽  
Adam Mead ◽  
Sonia Fox ◽  
Anesh Panchal ◽  
Christina Yap ◽  
...  
Keyword(s):  
Essential Thrombocythemia ◽  
Research Funding ◽  
Advisory Board ◽  
Symptom Improvement ◽  
Separate Analysis ◽  
Molecular Response ◽  
Molecular Responses ◽  
Early Satiety ◽  
Patient Reported ◽  
Grade 3

Abstract MAJIC is a phase II trial of Ruxolitinib (RUX) vs Best Available Therapy (BAT) in essential thrombocythemia (ET) patients with resistance/intolerance to Hydroxycarbamide (HC) per European LeukemiaNet (ELN) criteria. Primary outcome was rate of complete hematological response (CHR) within 1 year (ELN criteria); secondary outcomes included partial HR, safety, thrombosis, hemorrhage, progression free survival (including transformation), molecular response (MR), symptom & quality of life (QOL) assessment. We present new data concerning molecular, symptom & clinical responses. Patients were stratified by JAK2V617F status, patient-reported symptoms & QOL determined using EQ5D, MDASI & MPN Symptom Assessment Form (MPN10), & compared using linear mixed models of post-baseline scores through month 12 adjusting for baseline; response was defined as ≥50% reduction in MPN10 total symptom score (TSS). JAK2/CALR/MPL allele burdens were assessed at baseline & 4 monthly. 110 patients were eligible for the modified ITT analysis, 58 (52%) & 52 (48%) in RUX & BAT arms respectively, comprising 44 males, 66 females, mean age 64.2ys, & resistant (24.5%), intolerant (51.8%) or both (22.7%) to HC. CHR was achieved in 27 (46.6%) of RUX patients vs 23 (44.2%) BAT patients (χ2 test p= 0.81). PHR occurred in 26 (44.8%) & 27 (51.9%) of RUX & BAT treated respectively. Grade 3 or 4 anemia occurred in 19% & 0% for RUX arm vs 0% (both grades) for BAT arm, grade 3 or 4 thrombocytopenia in 5.2% & 1.7% of RUX vs 0% (both grades) of BAT patients respectively. Grade 3 or 4 infections occurred in 10.3% of RUX patients vs 3.6% BAT arm. 9 RUX treated patients had 10 thrombotic events & 1 RUX patient a hemorrhage; vs 5 thrombotic & 5 hemorrhagic events in BAT patients (adjusted following central review). Transformations to post-ET MF occurred in 8 RUX vs 3 BAT treated patients, 1 RUX patient developed AML. 2 non-treatment related deaths occurred in each arm. Mean MPN-10 TSS & individual symptoms of early satiety & itching during the first 12 months were all significantly lower for RUX vs BAT (all p<0.05). Patients who achieved CHR had significantly better TSS, fatigue, inactivity, concentration problems, & MDASI symptom interference (all p<0.05) at baseline vs those without; however, scores during treatment did not appear to differ between CHR & non CHR groups after adjusting for these baseline differences. Allele burden during study & MRs (per ELN-IWG criteria Barosi Blood 2013) are shown in Table 1. Assays for JAK2 V617F were performed independently in 3 centres using qPCR (Guy's), TSCA NGS (Oxford) & amplicon-based NGS (Salisbury) & revealed Mean (range): JAK2 (Guy's): 33.2 (0.1-94.6), N=52; JAK2 (Oxford): 38.0 (0.5-92.2), N=52; JAK2 (Sal): 38.3 (0-90.0), N=50 (limited to JAK2 positive only). With Interclass Correlation Coefficient as follows ICC (Guy's v Oxford): 0.92 (95% CI 0.86-0.95) ICC (Guy's v Sal): 0.92 (95% CI 0.86-0.95) ICC (Oxford v Sal): 0.997 (95% CI 0.994-0.998). Notably MRs (n=5) only occurred with RUX treatment. There was no pattern of MR or progression with C/PHR or transformation, but 1 patient who transformed to PET MF had a complete MR. In a separate analysis baseline symptoms & QOL were not associated with JAK2, CALR, nor MPL status. Within RUX, baseline symptoms & QOL did not predict MR; however, fatigue, early satiety & abdominal discomfort (all p<0.05, Table 1) were significantly lower among those with MR vs not during treatment with a descriptively higher symptom response rate (2/4 [50%] vs 9/30 [30%]). Three non pre-specified multivariate analyses were performed to assess baseline factors influencing CHR (modelled for: treatment received, HC resistance or intolerance, white cell count, platelets, Hb & JAK2/CALR status); occurrence of ≥ grade 3 anemia or thrombocytopenia (modelled for: Hb (≥ 100g/dl) JAK2/CALR status); & transformation to PET-MF (modelled for: treatment, Hb ≤100g/dl). Only baseline Hb ≤100g/dl was significant for grade 3+ anemia (OR [95% CI]=0.17 [0.04, 0.72]), & PET-MF only occurred in patients with baseline WBC <10x109/L. This updated analysis shows that HC resistant/intolerant ET is clinically & molecularly diverse. We confirm that these patients are at high risk of thrombosis & transformation as suggested in prior retrospective studies. Molecular responses were limited to RUX & for the first time we demonstrate such responses may correlate with symptom improvement but not always with progression events. Disclosures Harrison: Shire: Honoraria, Speakers Bureau; Gilead: Honoraria, Speakers Bureau; Incyte Corporation: Honoraria, Speakers Bureau; Baxaltra: Consultancy, Honoraria, Speakers Bureau; Novartis: Consultancy, Honoraria, Other: travel, accommodations, expenses, Research Funding, Speakers Bureau. Mead:Novartis: Honoraria, Research Funding, Speakers Bureau. Aliman:Novartis: Other: Institutional funding and grant for international conference. . Chen:Novartis: Other: Advisory Board. Coppell:Novartis: Other: Travel, accommodation and conference attendance. Knapper:ONO pharmaceuticals: Research Funding; Novartis: Honoraria, Other: Travel and expenses for international conferences. Ali:Novartis: Honoraria, Other: Conference sponsorship, advisory board meetings. Hamblin:Novartis: Other: Advisory Board. Dueck:Bayer: Honoraria. Cross:Novartis: Consultancy, Honoraria, Research Funding, Speakers Bureau. Mesa:Celgene: Research Funding; Promedior: Research Funding; CTI: Research Funding; Gilead: Research Funding; Incyte: Research Funding; Galena: Consultancy; Ariad: Consultancy; Novartis: Consultancy. McMullin:Novartis: Honoraria, Speakers Bureau.

scholarly journals Elipsis: A Longitudinal Study of Electronic Patient-Reported Outcomes, Actigraphy and Biomarkers to Identify and Assess at-Home Vaso-Occlusive Crises in Adults and Adolescents with Sickle Cell Disease

Blood ◽  
2017 ◽  
Vol 130 (Suppl_1) ◽  
pp. 973-973
Author(s):  
Michael U. Callaghan ◽  
Patrick C. Hines ◽  
Debra D Pittman ◽  
David Beidler ◽  
Denis Rybin ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Board Of Directors ◽  
Research Funding ◽  
Patient Reported Outcomes ◽  
Clinical Laboratory ◽  
Advisory Committees ◽  
Pain Scores ◽  
Patient Reported ◽  
Platelet Aggregates ◽  
At Home

Abstract Clinical trials in sickle cell disease (SCD) continue to have challenges achieving clinical endpoints. Most SCD clinical trials have focused on painful vaso-occlusive crisis (VOC) episodes because they are prevalent, debilitating and often lead to medical contact. However with VOC as a clinical endpoint: there are no objective, quantifiable biomarkers of pain; pain may not be specific to VOC; the threshold for medical contact varies between patients; VOCs occurring at home without medical contact are not captured; other components of VOC (e.g., fatigue, functioning) are poorly assessed. We therefore undertook the present non-interventional, longitudinal study to test novel tools for the identification of VOCs occurring in SCD patients with varying degrees of medical contact. During 6 months of evaluation, longitudinal measures of pain, fatigue, function, activity (by actigraphy), clinical laboratory and biomarker samples from SCD patients (+/- hydroxyurea therapy) in steady state to VOC were studied. A novel electronic patient-reported outcome tool (ePRO) enabled patients to self-report daily pain, fatigue, function, and medication use. It was also used to report VOC in real time, triggering an alert to a mobile phlebotomy team. Blood collections were taken within 24 and 48 hours of self-reported VOC onset (either at home, emergency department [ED], or hospital). Follow-up blood samples were collected 2 days after resolution of the VOC. Baseline blood samples were drawn at home every 3 weeks during stable, non-VOC periods. Biomarker assays included leukocyte-platelet aggregates and circulating microparticles measured by flow cytometry, cell adhesion in microfluidic flow-based assays to immobilized vascular cell adhesion molecule or P-selectin, and a panel of soluble adhesion molecules, cytokines, inflammatory mediators and coagulation factors. Patients wore a Phillips Actiwatch Spectrum™ actigraphy device to track sleep and activity. Patient-reported outcomes, activity, and biomarkers on non-VOC days were compared to those on VOC days using a mixed model approach and results are reported as means with 95% confidence intervals (95%CI). In this study 27 of 35 patients experienced a total of 286 days with VOC &gt;4 hr (VOC days), of which only 58 days (20%) resulted in healthcare utilization such as contacting provider, visiting ED and/or hospitalization. VOC days had significantly higher pain scores (scale: 0-10) with worst pain score increased by 4.5 (95% CI 4.3-4.7) compared to non-VOC days. VOCs requiring medical contact had significantly higher worst pain scores compared to at-home VOCs. Similar changes observed with reported least, average and pain right now. VOC days had significantly higher fatigue scores by 2.3 (95% CI 2.1-2.5) points (scale: 0-10). However, fatigue scores during at-home VOCs were not different from VOCs requiring medical contact. VOC days were associated with significantly decreased functional scores (physical, social, self-care and role activity), with significantly greater decreases during VOCs requiring medical contact compared to at-home VOCs. Different activity profiles were identified for non-VOC, at-home VOC and medical contact VOC days by actigraphy monitoring. At-home VOC days exhibited increased (34%, 95% CI 9%-64%) daytime resting compared to non-VOC days. Medical contact VOCs had decreased average and peak activity, and increased daytime resting compared to non-VOC days. A sleep fragmentation index trended up for both at-home (16%) and medical contact VOC days (18%). Significant changes during VOC days were observed in a subset of clinical laboratory and biomarker measures. Examples include: C-reactive protein (54% increase) and nucleated RBC (34% increase) in the clinical laboratory panel; monocyte-platelet aggregates (25% increase) and neutrophil-platelet aggregates (35% increase) in the biomarker panel. This innovative at-home study design demonstrates the feasibility of monitoring out of hospital pain and use of patient-reported VOC day as a potential endpoint for clinical trials. Electronic patient-reported outcomes, actigraphy and clinical laboratory and biomarkers may enable improved identification and assessment of at-home VOCs for further clinical studies. Disclosures Callaghan: CSL Behring: Membership on an entity's Board of Directors or advisory committees; Grifols: Membership on an entity's Board of Directors or advisory committees; Biogen: Membership on an entity's Board of Directors or advisory committees; Global Blood Therapeutics: Other: Site PI; Baxalta: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Roche/Genentech: Membership on an entity's Board of Directors or advisory committees, Other: Site PI, Speakers Bureau; Octapharma: Membership on an entity's Board of Directors or advisory committees; Alnylam Pharmaceuticals, Inc: Other: Owns stock, stock options, or bonds ; Shire: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Bayer HealthCare; Pfizer Inc.; Roche; Shire: Consultancy; Sancillio: Other: Site PI; Bayer: Membership on an entity's Board of Directors or advisory committees; Pfizer Inc.: Membership on an entity's Board of Directors or advisory committees, Other: Site PI, Research Funding; Roche; Shire: Speakers Bureau; Novo Nordisk: Speakers Bureau. Pittman: Pfizer: Employment. Beidler: Pfizer: Employment. Rybin: Pfizer: Employment. Liu: Functional Fluidics: Employment. Pleil: Pfizer: Employment. Barsdorf: Pfizer: Employment. David: Pfizer: Employment. Simmons: Pfizer: Employment. Frelinger: Baxalta: Research Funding; Ionis: Research Funding; Sysmex: Research Funding; Pfizer: Research Funding; Ironwood: Research Funding; GLSynthesis: Research Funding. Michelson: Eisai: Research Funding; Ionis: Research Funding; Ironwood: Research Funding; Pfizer: Research Funding; Sysmex: Research Funding; GLSynthesis: Research Funding; Janssen: Membership on an entity's Board of Directors or advisory committees; Instrumentation Laboratory: Consultancy; AstraZeneca: Membership on an entity's Board of Directors or advisory committees; Elsevier: Patents & Royalties; Baxalta: Research Funding. Clarke: Pfizer: Employment. Charnigo: Pfizer: Employment.

scholarly journals Emicizumab Outcomes in Hemophilia A Using Real-World Data from the Canadian Hemophilia Bleeding Disorder Registry

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 347-347
Author(s):  
Man-Chiu Poon ◽  
Adrienne Lee ◽  
Federico Germini ◽  
Arun Keepanasseril ◽  
Quazi Ibrahim ◽  
...  
Keyword(s):  
Stock Options ◽  
Research Funding ◽  
Patient Reported Outcomes ◽  
Hemophilia A ◽  
Severe Disease ◽  
Mild Disease ◽  
Patient Reported ◽  
Privately Held ◽  
Mcmaster University

Abstract Background: The Canadian Hemophilia Bleeding Disorders Registry (CBDR) is a clinical database including data from all Canadian Hemophilia Treatment Centers (HTCs) to assist in the management of hemophilia and other bleeding disorders. The CBDR launched on July 1, 2015 and integrates the data entry platform for patients, myCBDR, allowing direct entry of treatments, bleeding events, and other patient reported outcomes (PRO) data. Health Canada approved emicizumab in August 2018 for the treatment of persons with hemophilia A (PwHA) with factor (F)VIII inhibitors, and for PwHA without FVIII inhibitors in 2019, enabling some patients without FVIII inhibitors to be provided compassionate access beginning November 2019. Our aim for this analysis was to use the CBDR data to describe the demographics of the emicizumab-treated PwHA population and assess its effectiveness, safety, treatment patterns, and the impact on disease burden. Methods: De-identified data were extracted from the CBDR database for all registered PwHA who had received emicizumab at least once up to December 31, 2020. Disease severity is defined by the level of endogenous clotting FVIII activity. Effectiveness outcomes include number of patients (%) with zero treated bleeds, joint bleeds, and spontaneous bleeds; annualized bleeding rates (ABR) for any bleeds and treated bleeds; Hemophilia Joint Health Score (HJHS); Patient Reported Outcomes Burdens and Experiences (PROBE); and EQ-5D-5L index and visual analog scale (VAS). ABRs were calculated as (total number of bleeds/duration of follow-up [days])*365.25. All analyses were performed based on the observed values, without imputation. This study was approved by the research ethics board of McMaster University and other participating centers, and abides by the guiding principles of the Declaration of Helsinki. Results: 73 PwHA who received emicizumab at least once up to December 31, 2020 were identified. Demographic characteristics, severity, inhibitor status, and treatments are described in Table 1. Median (IQR) age for the entire cohort was 19.7 (10.0, 40.6) years with 45.2% ≤18 years. There were 64 PwHA with severe disease, 7 with moderate disease and 2 with mild disease; both cases with mild disease had current FVIII inhibitors. 49 PwHA had current FVIII inhibitors, 12 had a history of FVIII inhibitors, and 12 had no FVIII inhibitors. 5/73 (6.8%) received immune tolerance induction (ITI) treatment while on emicizumab. Two cases of rash (allergic or acute reactions) were reported (2/73, 2.7%) of which one (reported 6 days after administration) was possibly related to emicizumab according to the reporting HTC. No thromboembolisms or thrombotic microangiopathies were observed. Median ABR (IQR) for the entire study population was 0.0 (0, 0) and 59/73 (80.8%) had no recorded bleeds. In the 14/73 (19.2%) with recorded bleeds, median ABR (IQR) was 2 (1, 3); 8 of those 14 had joint bleeds and 7 had spontaneous bleeds. HJHS was recorded for 23, PROBE Score for 9, EQ-5D-5L for 9 and EQ-5D-5L VAS for 4 PwHA (Table 2). Conclusions: The Canadian population treated with emicizumab had severe disease and current or historical FVIII inhibitors. The bleed outcomes are consistent with earlier publications, showing 80.8% had no recorded bleeds. The CBDR will allow for longitudinal follow-up of this patient population. Our results can inform healthcare practitioners and regulatory authorities on the real-world safety and effectiveness outcomes of emicizumab in PwHA with and without FVIII inhibitors. Figure 1 Figure 1. Disclosures Poon: Roche: Honoraria; Pfizer: Honoraria; Novo Nordisk: Honoraria; Bioverativ/Sanofi: Honoraria; CSL-Behring: Honoraria, Research Funding; Bayer: Honoraria, Research Funding; University of Calgary: Current Employment; Takeda: Honoraria. Lee: Roche: Honoraria; Pfizer: Honoraria, Speakers Bureau; Novo Nordisk: Honoraria, Speakers Bureau; CSL Behring: Honoraria; Biovertiv/Sanofi: Honoraria, Research Funding; Bayer: Research Funding, Speakers Bureau; Takeda: Honoraria. Keepanasseril: McMaster University: Current Employment; NovoNordisk Canada: Consultancy. Ibrahim: McMaster University: Current Employment. Nissen: F. Hoffmann-La Roche Ltd: Current Employment, Current holder of stock options in a privately-held company; Novartis: Consultancy; Actelion: Consultancy. Sanabria: F. Hoffmann-La Roche Ltd: Current Employment, Current holder of individual stocks in a privately-held company. Santos: F. Hoffmann-La Roche Ltd.: Current Employment; Roche: Current holder of individual stocks in a privately-held company, Current holder of stock options in a privately-held company; Baxter: Current holder of individual stocks in a privately-held company, Current holder of stock options in a privately-held company; Takeda: Current holder of individual stocks in a privately-held company, Current holder of stock options in a privately-held company. Iorio: McMaster University: Current Employment; Bayer (funding to the institution): Research Funding; BioMarin (funding to the institution): Research Funding; NovoNordisk (funding to the institution): Research Funding; Octapharma (funding to the institution): Research Funding; Pfizer (funding to the institution): Research Funding; Roche (funding to the institution): Research Funding; Sanofi (funding to the institution): Research Funding; Sobi (funding to the institution): Research Funding; Takeda (funding to the institution): Research Funding.

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