Patient-reported quality of life (QOL) following CTL019 in pediatric and young adult patients (pts) with relapsed/refractory (r/r) b-cell acute lymphoblastic leukemia (B-ALL).

2017 ◽  
Vol 35 (15_suppl) ◽  
pp. 10523-10523 ◽  
Author(s):  
Andrew Charles Dietz ◽  
Stephan A. Grupp ◽  
Theodore Willis Laetsch ◽  
Heather Stefanski ◽  
Gary Douglas Myers ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Young Adult ◽  
Complete Blood Count ◽  
Lymphoblastic Leukemia ◽  
Complete Response ◽  
Adverse Event Rate ◽  
Cell Transplant ◽  
Patient Reported ◽  
Vas Scores

10523 Background: The global ELIANA trial (NCT02435849) evaluates the efficacy and safety of CTL019, a single infusion of genetically modified autologous chimeric antigen receptor–expressing T cells targeting CD19+ cells in pediatric and young adult r/r B-ALL pts. Analyses show a complete response rate of 82% with or without complete blood count recovery ≤ 3 months. A serious adverse event rate of 71% was observed in ≤ 8 weeks of infusion, decreasing to 17% at > 8 weeks (Grupp S, et al. Blood. 2016;128(22) [abstract 221].). This analysis further evaluates the clinical benefit of CTL019. QOL was assessed before and after CTL019 infusion. Methods: Infused pts were 3-23 y/o with CD19+ B-ALL who were chemo refractory, relapsed after allogeneic stem cell transplant (SCT), or otherwise ineligible for SCT. Pts ≥ 8 y completed the Pediatric Quality of Life Inventory (PedsQL) and EuroQol EQ-5D at baseline and following CTL019 infusion. Minimal clinically important differences are estimated to be 4.4 and 7 to 10 for PedsQL and EQ-5D, respectively. Results: 62 of 81 enrolled pts were infused; 56% had relapsed after SCT with median of 3 prior therapies. At interim analysis, 50 pts were treated ≥ 3 months prior to data cutoff and eligible for primary efficacy analysis. A total of 39 pts were ≥ 8 y. Mean PedsQL total and EQ-5D VAS scores, respectively, were 58.4 and 69.4 at baseline. Mean changes from baseline for the PedsQL total and EQ VAS scores, respectively, were 13.9 and 13.7 at month 3 and 12.8 and 10.9 at month 6, supporting clinically meaningful improvements in QOL. Similar trends were observed with each PedsQL subscale. With EQ-5D, the proportions of pts reporting problems with mobility, self-care, usual activities, anxiety/depression, or pain/discomfort were notably decreased at months 3 and 6 compared with baseline. Conclusions: Clinically meaningful improvements in QOL were observed at 3 and 6 months after CTL019 therapy in pediatric and young adult r/r B-ALL pts, including fewer problems in each EQ-5D domain. These results suggest improved QOL after this one-time immunocellular therapy beyond the period of acute toxicities. Clinical trial information: NCT02435849.

Feasibility of olanzapine at reduced dose in highly emetogenic chemotherapy: A randomised controlled trial against aprepitant in triple therapy (FORESIGHT).

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. 12087-12087
Author(s):  
Michelle Chen ◽  
Isabelle Baron ◽  
Stephanie Beaulieu ◽  
Annick Dufour ◽  
Nathalie Letarte ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Large Scale ◽  
Controlled Trial ◽  
Complete Response ◽  
Research Centre ◽  
Highly Emetogenic Chemotherapy ◽  
Patient Reported ◽  
Delayed Phase ◽  
Drug Prophylaxis

12087 Background: Olanzapine is used as an adjunct antiemetic in oncology as salvage therapy and in four-drug prophylaxis. Growing literature supports its effectiveness in initial three-drug prophylaxis in highly emetogenic chemotherapy (HEC). Methods: This prospective, multi-centre, open-label study evaluated the feasibility of a large-scale randomized controlled trial comparing the effectiveness and tolerability of 5 mg olanzapine once daily for four days (starting the night before chemotherapy) versus standard dose aprepitant (in tritherapy with standard ondansetron and dexamethasone) in treatment-naive patients receiving the first cycle of a HEC. Secondary outcomes included: complete response (no nausea, no emesis, no use of rescue medication), complete remission (no emesis, no rescue medication), intensity of patient-reported nausea and emesis on a visual analog scale, quality of life (scored with the Functional Living Index Emesis [FLIE]), and incidence of adverse events. Results: We randomized 30 patients in an intent-to-treat analysis. The large-scale trial was deemed not feasible without support from a research centre. Complete response rates were significantly higher in the olanzapine group in the delayed phase (24-120h post-chemotherapy) (86,7% v 21,4%, p < 0,001) and overall phase (0-120h post-chemotherapy) (60,0% v 21,4%, p = 0,04). Similar results were observed for complete remission. Intensity of patient-reported nausea was significantly lower in the olanzapine group in the delayed phase (p = 0,001). FLIE scores were significantly lower for the nausea domain (mean 62,3 v 60,9, p = 0,004) and overall score (124,3 v 108,8, p = 0,006). Depression on the ESAS-R was more common in the aprepitant group (0% v 38%, p = 0,01). Other adverse events were not significantly different. Conclusions: Support from a research centre must be ensured for study feasibility. Tritherapy olanzapine significantly improved complete response and remission in the delayed and overall phases post-chemotherapy among patients receiving HEC. It was also associated with higher quality of life and a reassuring safety profile. This feasibility trial, despite its small sample size, is one of the first prospective randomised trials to suggest similar efficacy of 5 mg olanzapine to aprepitant and to measure a difference in patient quality of life with this regimen. Clinical trial information: NCT04075955 .

Functional and Quality of Life Outcomes of Surgery for Degenerative Cervical Myelopathy: a Quality Improvement Study

2020 ◽  
Author(s):  
Babak Mirzashahi ◽  
Pejman Mansouri ◽  
Arvin Najafi ◽  
Saeed Besharati ◽  
Mohammad Taha Kouchakinejad ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Cervical Myelopathy ◽  
Short Form ◽  
Sf 36 ◽  
Patient Reported ◽  
Vas Scores ◽  
One Year ◽  
Degenerative Cervical Myelopathy

Background: This study aimed to determine the outcome of surgical treatments in patients with degenerative cervical myelopathy (DCM). During one-year follow-up period, we evaluated patient-reported functional and quality of life (QOL) measures.   Methods: In a retrospective single-center study, we collected data of patients with DCM who underwent cervical fusion surgeries in Imam Khomeini Hospital, Tehran, Iran, from 2011 to 2015. Patients underwent single or multi-level anterior cervical discectomy and fusion (ACDF), anterior cervical corpectomy and fusion (ACCF), or posterior laminectomy and fusion. We utilized patient-reported assessments including Short Form 36 (SF-36), Visual Analogue Scale (VAS), Neck Disability Index (NDI), and Nurick grade. Follow-up was performed at 6 weeks, 3 months, 6 months, and 12 months post-operatively to assess the outcome of surgery.   Results: Ninety patients (56 men, 34 women) with a mean age of 54.1 (27-87) years were included. Comparison of pre- and post-operative scores showed significant improvement in SF-36 parameters, VAS, NDI, and Nurick grade (P < 0.001). Also, women’s VAS scores improved more than men's VAS scores during the follow-up period (P < 0.050). Age and type of surgery did not significantly affect the SF-36 parameters, VAS, NDI, and Nurick grade (P > 0.05).   Conclusions: Cervical surgeries in patients with different severity of DCM can improve different aspects of QOL during one-year after surgery

scholarly journals An Observational Study to Evaluate the Efficacy and Quality of Life Provided by Netupitant and Palonosetron Regimen against Ondansetron in Management and Prevention of CINV

2021 ◽  
Vol 68 (2) ◽  
Author(s):  
Meghana Dutta ◽  
Rooha K
Keyword(s):  
Quality Of Life ◽  
Treatment Plan ◽  
Complete Response ◽  
Nausea And Vomiting ◽  
Health Concern ◽  
Patient Reported ◽  
Group A ◽  
Group B ◽  
The Impact

Cancer is a public health concern amongst millions of humans and claims hundreds of lives every year. The maximum worry-inducing side effect of cancer treatment is nausea and vomiting. Therefore, stopping and managing chemotherapy-induced nausea and vomiting is an important part of a cancer patient’s treatment plan. In this study, we evaluated the efficacy and quality of life provided by two commonly used antiemetic regimens in the management and prevention of chemotherapy-induced nausea and vomiting (CINV) in cancer patients. We assessed patient-reported nausea, vomiting, use of rescue medication, and Functional Living IndexEmesis (FLIE) questionnaire results, and used them as parameters to make comparisons. We also examined the percentage of patients showing complete response (CR; no emesis and non-use of rescue antiemetics), and the impact of CINV on patient’s daily life during the acute and delayed phases. The results show that the complete response is achieved by 26 patients in group-B and 18 patients in group-A, from the total 60 patients, while the FLIE scores indicated better quality of life is maintained in group-B (76.6%). In the study, the predominance of Netupitant and Palonosetron regimen to Ondansetron was demonstrated.

Patient-reported Health-related Quality of Life with Apremilast for Psoriatic Arthritis: A Phase II, Randomized, Controlled Study

2013 ◽  
Vol 40 (7) ◽  
pp. 1158-1165 ◽  
Author(s):  
Vibeke Strand ◽  
Georg Schett ◽  
ChiaChi Hu ◽  
Randall M. Stevens
Keyword(s):  
Quality Of Life ◽  
Psoriatic Arthritis ◽  
Health Related Quality ◽  
Randomized Controlled ◽  
Sf 36 ◽  
Patient Reported ◽  
Related Quality ◽  
Health Related ◽  
Vas Scores

Objective.Apremilast, a specific inhibitor of phosphodiesterase 4, modulates proinflammatory and antiinflammatory cytokine production. A phase IIb randomized, controlled trial (RCT) evaluated the effect of apremilast on patient-reported outcomes (PRO) in psoriatic arthritis (PsA).Methods.In this 12-week RCT, patients with active disease (duration > 6 mo, ≥ 3 swollen and ≥ 3 tender joints) received apremilast (20 mg BID or 40 mg QD) or placebo. PRO included pain and global assessment of disease activity [visual analog scale (VAS)], Health Assessment Questionnaire-Disability Index (HAQ-DI), Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F), and Medical Outcomes Study Short-Form 36 Health Survey (SF-36) assessing health-related quality of life (HRQOL). Percentages of patients reporting improvements ≥ minimum clinically important differences (MCID) and correlations between SF-36 domains and pain VAS, HAQ-DI, and FACIT-F were determined.Results.Among the 204 randomized patients (52.5% men; mean age 50.6 yrs), baseline SF-36 scores reflected large impairments in HRQOL. Apremilast 20 mg BID resulted in statistically significant and clinically meaningful improvements in physical and mental component summary scores and 7 and 6 SF-36 domains, respectively, compared with no change/deterioration in placebo group. Patients receiving apremilast 20 mg BID and 40 mg QD reported significant improvements ≥ MCID in global VAS scores and FACIT-F versus placebo, and significant improvements in pain VAS scores. Moderate-high, significant correlations were evident between SF-36 domains and other PRO.Conclusion.Apremilast resulted in statistically significant and clinically meaningful improvements in HRQOL, pain and global VAS, and FACIT-F scores.

AL Amyloidosis and Patient Reported Quality of Life

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 3317-3317
Author(s):  
Rahma Warsame ◽  
Shaji Kumar ◽  
Carrie A. Thompson ◽  
Morie A. Gertz ◽  
Martha Q. Lacy ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Research Funding ◽  
Early Death ◽  
Cell Transplant ◽  
Significant Difference ◽  
Patient Reported ◽  
One Year ◽  
Over Time

Abstract Background: Patients with light chain amyloidosis (AL) often have delayed diagnosis and present with significant symptomatology; this may result in decreased quality of life (QOL). With improving treatment options providing longer survival, it is increasingly important to assess QOL. However there is paucity of data in the literature addressing QOL in AL patients. We prospectively employ a "Hematology Patient Reported Symptom Screen" (HPRSS) which consists of three questions about fatigue, pain and overall QOL. The aim of this study is to understand if HPRSS parameters predict various clinical outcomes. Methods: Eligibility for this retrospective study was as follows: 1) New diagnosis of AL between 2009-2014; 2) baseline HPRSS documented in the medical record; and 3) at least a year of follow-up, which included either death within or follow-up through 12 months after diagnosis. The HPRSS questions were rated on a 1-10 scale, with 10 being the worst for fatigue and pain, and 10 being the best for overall QOL. Scores were abstracted from visits at time of diagnosis, and at 12 months +/- 1 month post-diagnosis. We considered a 2-point difference in serial scores a "change" over time. Results: For the 302 patients in this study, the baseline median scores [interquartile range] for fatigue, pain, and QOL were 6 [3,7], 2 [0,5] and 5 [3,8], respectively. Median overall survival (OS) was 39.1 months, with 102 deaths in the first year. There were significant differences in baseline HPRSS between those who lived longer than one year and the early death patients in the domains of fatigue (5 [IQR 3, 7] vs. 7 [IQR 5, 8], p<0.0001) and QOL (6 [IQR 4, 8] vs. 5 [IQR 3, 7], p=0.006), but not in pain (2 [IQR 0, 5] vs. 2 [IQR 0, 5]). There were significant baseline differences in the early death group for alkaline phosphatase, bilirubin, creatinine, and Mayo stage. Patients who received ASCT had the best baseline fatigue 4 [2.5,6] and QOL 7 [5,8] scores and were significantly different from those who did not receive ASCT [fatigue p<0.0001) and QOL (p<0.0001)] On univariate analysis fatigue and QOL were prognostic for OS. On multivariate analysis Mayo 2012 staging, autologous stem cell transplant and baseline fatigue remained independently prognostic. When analyses were restricted to the 125 patients with HPRSS measurements at 12 months, we found that over time QOL scores improved significantly 6 [IQR 3.5, 8] to 7 [IQR 5, 8] (two sided Wilcoxon signed rank p=0.01), but fatigue (5 [IQR 2, 5] to 4.5 [IQR 3, 6]) and pain scores (2 [IQR 0 ,4] to 1.5 [IQR 0, 4]) did not. Patients with worse baseline parameters tended to have improvement in QOL by 12 months while those with the best baseline parameters tended to decline in QOL although not statistically significant. When we included the 102 patients who died within 12 months to the comparison, the early death patients had the worst baseline parameters and there were significant differences across all 4 groups for most characteristics (Table 1). There was no association between achieving hematologic or organ response with change in QOL. Conclusion: Asking patients with AL to rate their fatigue and QOL using a 10-point scale has predictive value. Patient reported fatigue at diagnosis is an independent prognostic factor for survival. Survival at one year was associated with significant improvement in QOL. Table 1. Baseline parameters between patients with early death and/or survive 12 months. Grouped by Patient Reported QOL at 12 months Relative to Baseline Dead by 12 months, n=102 Improved, n=44 Stable, n=55 Worsened, n=26 pa HPRSS scores [IQR] Baseline QOL Baseline fatigue Baseline pain 12 month QOL 12 month fatigue 12 month pain 5 [3,7] 7 [5,8] 2 [0,5] NA NA NA 4 [3-5] b,c,d6 [4-8] b,c2 [0-4] 7 [6-9] b,d4 [1.25-6] 1 [0-3] 7 [4-8] 4 [2-7] 2 [1-5] 7 [4-8] 5 [3-6] 2 [0-4] 8 [7-10] 2.5 [0-5.25] 2 [0-6.25] 5 [3-7] 5 [3-6] 2.5 [1-4] <0.0001 <0.0001 NS NS 0.001 NS dFLC, mg/dL 48 (2.89-726) 30 (1.1-494) 19 (36-455) 22 (0.2-2097) 0.008 Troponin, ng/mL 0.065 (<0.01-0.84) 0.02 (<0.01-1.6) 0.01 (<0.01-0.19) 0.01 (<0.01-0.14) <0.0001 NT-proBNP pg/mL 5222 (159-70,000) 1766 (26-16868) 1381 (24-19180) 496 (56-2973) <0.0001 Received Transplant (%) 4 (4) 13 (33) 36 (50) 13 (50) 0.0002 a Significant by Wilcoxon across all 4 categories; b Significant difference between Improved and Worsened; c Significant difference between Improved and Stable; d Significant difference between Worsened and Stable Disclosures Kumar: Millenium: Consultancy, Research Funding; Celgene: Consultancy, Research Funding; Abbvie: Research Funding; Janssen: Consultancy, Research Funding; BMS: Consultancy, Research Funding; Novartis: Research Funding; Onyx: Consultancy, Research Funding. Thompson:Kite Pharma: Research Funding.

scholarly journals Long-Term Quality of Life after Hematopoietic Cell Transplantation for Sickle Cell Disease: A Preliminary Report from the Sickle Cell Transplant Evaluation of Long Term and Late Effects Registry (STELLAR)

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 425-425
Author(s):  
Staci D. Arnold ◽  
Nitya Bakshi ◽  
Zhao Chang ◽  
Diana Ross ◽  
Allistair Abraham ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Sickle Cell ◽  
Univariate Analysis ◽  
Cell Transplant ◽  
Significant Difference ◽  
Patient Reported ◽  
Ed Visits ◽  
The Mean

Abstract Introduction: Sickle cell disease (SCD) is characterized by unpredictable, recurrent acute and chronic persistent pain, and progressive organ damage leading to substantial morbidity, impaired quality of life and increased healthcare utilization. Hematopoietic cell transplantation (HCT) is the only therapy for SCD with curative intent. HCT is associated with excellent overall and event free survival especially in young children undergoing HCT from HLA identical family donors. Given these outcomes and improved supportive care, HCT is increasingly being offered to adults with SCD. However, a paucity of data exists on health related quality of life (HrQoL) in patients who are long term survivors of HCT. Therefore, a clear need exists to study HrQoL in children and adults post HCT to determine the long term benefit of HCT for SCD. We report the results of an interim analysis of HrQoL in recipients of HCT for SCD. Methods: We enrolledchildren and adults with SCD ≥ 1-year post-HCT in the Sickle Cell Transplant Evaluation of Long Term and Late Effects Registry (STELLAR). We obtained patient self-report of health status and health practices using a web based platform. Individuals under age 18 years and/or proxies completed Patient Reported Outcome Measurement Information System (PROMIS) version 25. Individuals over age 18 completed PROMIS version 29. Surveys were completed at enrollment and annually thereafter. T-scores were calculated for each survey domain. HrQoL in children (PROMIS25) and adults (PROMIS29) was compared to age appropriate norms (mean 50 ± 10). Univariate analysis was performed by age, gender, marital status, time from transplant, history of preHCT stroke, frequency of pain, number of emergency department (ED) visits, length of stay, income, and education assessed at the time of PROMIS survey administration. Statistical analysis was conducted using SAS Version 9.4. Descriptive statistics for each variable were reported. For numeric covariates, the mean and standard deviation (SD) were calculated and presented with frequency and its percentage shown for category variables. For repeated measures, generalized estimating equation (GEE) model was performed to detect the association. The significance level was set at 0.05. Results: Twenty-seven individuals completed 30 PROMIS25 surveys and 15 individuals completed 19 PROMIS29 surveys from 2017-2021. The mean age at transplant was 11.4years (SD 3.1) and 23.8 years (SD 6.2) in adults, respectively. Sixty percent and 50%, respectively, were female. The mean time from transplant was 4.2 years (SD 3.9) and 9.1 years (SD 7.1), respectively. Physical function/mobility, anxiety, depression, fatigue, and pain T-scores for each PROMIS measure are reported below in Table 1. Univariate analysis of PROMIS25 T-scores showed a statistically significant difference in anxiety by age and peer relationships by age, gender, and time from transplant. Time from transplant also had a statistically significant association with pain interference. PROMIS29 T-scores also showed a statistically significant difference in anxiety by age. Gender had a statistically significant association with depression and fatigue. Number of ED visits had a significant association with physical function, depression, social participation, and pain interference (Table 2). Other covariates lacked a statistically significant association or had insufficient sample sizes for comparison. Conclusions: Long-term HrQoL after HCT for SCD is within population norms for adults and children, though children have better HRQoL compared to adults. Better scores in females suggests the need for study of gender as a mediator of HrQoL outcomes. The observed relationship between HrQoL domains and education, income, time from transplant, and number of ED visits, a surrogate for healthcare utilization, suggest the need for careful study of these and other factors that can influence HrQoL post HCT over the long-term. In addition, further research in patient reported outcomes has the potential to identify needed areas of intervention and surveillance for this population. Figure 1 Figure 1. Disclosures Guilcher: Project Sickle Cure Study: Other: Principal Investigator, Research Funding; BlueBirdBio: Research Funding.

scholarly journals Specialist cancer services for teenagers and young adults in England: BRIGHTLIGHT research programme

2021 ◽  
Vol 9 (12) ◽  
pp. 1-82
Author(s):  
Rachel M Taylor ◽  
Lorna A Fern ◽  
Julie Barber ◽  
Faith Gibson ◽  
Sarah Lea ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Young Adults ◽  
Young Adult ◽  
Health Care Professionals ◽  
Specialist Care ◽  
Specialist Services ◽  
Patient Reported ◽  
Treatment Centre ◽  
Teenagers And Young Adults

Background When cancer occurs in teenagers and young adults, the impact is far beyond the physical disease and treatment burden. The effect on psychological, social, educational and other normal development can be profound. In addition, outcomes including improvements in survival and participation in clinical trials are poorer than in younger children and older adults with similar cancers. These unique circumstances have driven the development of care models specifically for teenagers and young adults with cancer, often focused on a dedicated purpose-designed patient environments supported by a multidisciplinary team with expertise in the needs of teenagers and young adults. In England, this is commissioned by NHS England and delivered through 13 principal treatment centres. There is a lack of evaluation that identifies the key components of specialist care for teenagers and young adults, and any improvement in outcomes and costs associated with it. Objective To determine whether or not specialist services for teenagers and young adults with cancer add value. Design A series of multiple-methods studies centred on a prospective longitudinal cohort of teenagers and young adults who were newly diagnosed with cancer. Settings Multiple settings, including an international Delphi study of health-care professionals, qualitative observation in specialist services for teenagers and young adults, and NHS trusts. Participants A total of 158 international teenage and young adult experts, 42 health-care professionals from across England, 1143 teenagers and young adults, and 518 caregivers. Main outcome measures The main outcomes were specific to each project: key areas of competence for the Delphi survey; culture of teenagers and young adults care in the case study; and unmet needs from the caregiver survey. The primary outcome for the cohort participants was quality of life and the cost to the NHS and patients in the health economic evaluation. Data sources Multiple sources were used, including responses from health-care professionals through a Delphi survey and face-to-face interviews, interview data from teenagers and young adults, the BRIGHTLIGHT survey to collect patient-reported data, patient-completed cost records, hospital clinical records, routinely collected NHS data and responses from primary caregivers. Results Competencies associated with specialist care for teenagers and young adults were identified from a Delphi study. The key to developing a culture of teenage and young adult care was time and commitment. An exposure variable, the teenagers and young adults Cancer Specialism Scale, was derived, allowing categorisation of patients to three groups, which were defined by the time spent in a principal treatment centre: SOME (some care in a principal treatment centre for teenagers and young adults, and the rest of their care in either a children’s or an adult cancer unit), ALL (all care in a principal treatment centre for teenagers and young adults) or NONE (no care in a principal treatment centre for teenagers and young adults). The cohort study showed that the NONE group was associated with superior quality of life, survival and health status from 6 months to 3 years after diagnosis. The ALL group was associated with faster rates of quality-of-life improvement from 6 months to 3 years after diagnosis. The SOME group was associated with poorer quality of life and slower improvement in quality of life over time. Economic analysis revealed that NHS costs and travel costs were similar between the NONE and ALL groups. The ALL group had greater out-of-pocket expenses, and the SOME group was associated with greater NHS costs and greater expense for patients. However, if caregivers had access to a principal treatment centre for teenagers and young adults (i.e. in the ALL or SOME groups), then they had fewer unmet support and information needs. Limitations Our definition of exposure to specialist care using Hospital Episode Statistics-determined time spent in hospital was insufficient to capture the detail of episodes or account for the variation in specialist services. Quality of life was measured first at 6 months, but an earlier measure may have shown different baselines. Conclusions We could not determine the added value of specialist cancer care for teenagers and young adults as defined using the teenage and young adult Cancer Specialism Scale and using quality of life as a primary end point. A group of patients (i.e. those defined as the SOME group) appeared to be less advantaged across a range of outcomes. There was variation in the extent to which principal treatment centres for teenagers and young adults were established, and the case study indicated that the culture of teenagers and young adults care required time to develop and embed. It will therefore be important to establish whether or not the evolution in services since 2012–14, when the cohort was recruited, improves quality of life and other patient-reported and clinical outcomes. Future work A determination of whether or not the SOME group has similar or improved quality of life and other patient-reported and clinical outcomes in current teenage and young adult service delivery is essential if principal treatment centres for teenagers and young adults are being commissioned to provide ‘joint care’ models with other providers. Funding This project was funded by the National Institute for Health Research (NIHR) Programme Grants for Applied Research programme and will be published in full in Programme Grants for Applied Research; Vol. 9, No. 12. See the NIHR Journals Library website for further project information.

scholarly journals “I have SMA, SMA doesn’t have me”- A Qualitative Snapshot into the Challenges, Successes, and Quality of Life of Adolescents and Young Adults with SMA

2020 ◽  
Author(s):  
Allison Joy Mazzella ◽  
Mary Curry ◽  
Lisa Belter ◽  
Rosangel Cruz ◽  
Jill Jarecki
Keyword(s):  
Quality Of Life ◽  
Young Adults ◽  
Young Adult ◽  
Transition Period ◽  
Muscular Atrophy ◽  
Adolescents And Young Adults ◽  
Adolescent And Young Adult ◽  
Free Text ◽  
Patient Reported

Abstract Background: With the approval of three treatments for spinal muscular atrophy (SMA) and several promising therapies on the horizon, the SMA adolescent and young adult populations are expected to evolve in the coming years. It is imperative to understand this cohort as it exists today to provide optimal care and resources, as well as to assess possible treatment effects over time. In 2018, Cure SMA launched two initiatives geared towards understanding adolescents and young adults with SMA, ages 12-25. First, Cure SMA launched a Quality of Life (QoL) survey to capture quantitative and qualitative information on this specific age demographic. Concurrently, Cure SMA invited SMA-affected individuals, ages 12-25, to create a three-minute video on their everyday experiences living with SMA. An inductive thematic analysis of the free-text survey questions along with the video contest findings are reported here. Results: Eighty-five individuals — 6 type Is, 58 type IIs, and 21 type IIIs — completed the Quality of Life free-response, while six individuals participated in the SMA awareness video contest. In both settings, individuals detailed a variety of challenges, including but not limited to forming or maintaining close relationships, experiencing feelings of isolation, challenges with accessibility, independence, and dealing with the stigma of being perceived as mentally disabled. Individuals also discussed their successes, including but not limited to higher education enrollment and attendance, development of quality friendships, and perseverance through obstacles. Additionally, notably in the survey, 39% of respondents requested the creation of an SMA peer support group in efforts to connect with each other as well as collectively navigate the aforementioned challenges they face. Conclusion: Together, these findings provide a rare glimpse into the unique mindsets, challenges and motivations of SMA adolescents and young adults, via patient-reported measures instead of caregiver proxy. The adolescent and young adult age demographics assessed represent a critical transition period in life and in SMA care. No one understands the needs of an adolescent or young adult with SMA better than the individuals themselves, and it is critical to encapsulate their insights to affect change.

scholarly journals Mobile Phone Application Intervention for Coping with Cancer as a Young Adult: Feasibility Study (Preprint)

2020 ◽  
Author(s):  
Hanneke Poort ◽  
Annelise Ryan ◽  
Katelyn MacDougall ◽  
Paige Malinowski ◽  
Anna MacDonald ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Young Adult ◽  
Emotional Support ◽  
Self Efficacy ◽  
Qualitative Interviews ◽  
Pilot Trial ◽  
Smartphone Application ◽  
Coping With Cancer ◽  
Patient Reported

BACKGROUND Many young adult (YA) patients do not receive adequate psychosocial services to help them cope with cancer. OBJECTIVE To assess the feasibility and acceptability of a smartphone application (“iaya”) intervention that was designed to create an engaged community of YA patients and help them learn emotional coping skills. METHODS For this single-group pilot trial, 25 YA patients aged 18-39 years who were receiving active cancer treatment were asked to use the “iaya” app for 12 weeks. To collect app usage data, we used Mixpanel, an analytics platform for apps. Feasibility was assessed through rates of app sessions and coping exercises engaged, and intervention acceptability was evaluated with an app usability questionnaire and through qualitative interviews at study completion. We collected patient-reported outcome (PRO) data at baseline and at week 12 to explore self-efficacy for coping with cancer, self-efficacy for managing emotions, perceived emotional support, and quality of life. RESULTS Baseline PRO data indicated that participants scored relatively low on perceived emotional support, but reasonably high on self-efficacy for coping with cancer and managing emotions, as well as quality of life. Participants had a mean of 13 app sessions (SD=14) and 2 coping exercises engaged (SD=3.83) in 12 weeks. Only 8.7% of participants met our combined feasibility definition of ≥ 10 app sessions and ≥ 3 copings skills from different categories. Participants’ mean usability score was 73.7% (SD, 10.84), exceeding our predefined threshold of ≥ 70%, and qualitative feedback was generally positive. CONCLUSIONS While perceived acceptable by patients, the “iaya” smartphone app did not meet the a priori feasibility criteria as a stand-alone app intervention. Future studies should screen participants for unmet coping needs and consider integrating the app as part of YA patients’ psychosocial care. CLINICALTRIAL ClinicalTrials.gov NCT04119869.

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