scholarly journals Validation of a Modified Version of the Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 26-26
Author(s):  
Blake T. Langlais ◽  
Heidi E. Kosiorek ◽  
Gina L. Mazza ◽  
Carolyn Mead-Harvey ◽  
Richard Butterfield ◽  
...  
Keyword(s):  
Clin Oncol ◽  
Symptom Score ◽  
Internal Consistency ◽  
Research Funding ◽  
Symptom Assessment ◽  
Mean Difference ◽  
Total Symptom Score ◽  
Symptom Experience ◽  
Cronbach's Alpha ◽  
Highly Correlated

Background Patients with myeloproliferative neoplasms (MPNs) are faced with severe disease-related fatigue among a range of other constitutional and spleen-related symptoms. The MPN-Symptom Assessment Form (SAF) is recommended for use to characterize symptom burden (Scherber R, et al. Blood 2011). Within the SAF, a profile of 18 symptom items are evaluated ranging in severity from 0 (absent) to 10 (worst imaginable). The SAF is often summarized to the MPN-SAF Total Symptom Score (TSS) for analysis purposes - a single computed sum of the 10 most clinically meaningful symptom scores, including fatigue (Emanuel R, et al. J Clin Oncol 2012). Though the SAF includes a fatigue item, initial deployments of the MPN-SAF TSS incorporated a 0-10 scaled fatigue item taken from the Brief Fatigue Inventory (BFI; Mendoza T, et al. Cancer 1999). A subsequent version of the MPN-SAF TSS for use within myelofibrosis clinical trials (called the MFSAF v4; Gwaltney C, et al. Leuk Res 2017) employed a harmonized fatigue item. This analysis employing data from two studies was carried out to assess the use of the SAF fatigue item within the MPN-SAF TSS for consistency with the MFSAF v4. Methods Both BFI and SAF fatigue items were included in an initial online survey evaluating disease burden among patients with MPNs. Participants were assigned to survey variants as a function of their age. Survey variants included those to receive 1 instance of either the BFI or SAF fatigue item, instances of both BFI and SAF, or 2 instances of the same fatigue item. Surveying was aimed to assess the worst symptom experience in the patient's last 24 hours. Additionally, an independent survey assessing the impact of COVID-19 among MPN patients was deployed using the SAF fatigue item for the MPN-SAF TSS. This modified version was then used to test internal validity. Pearson correlation (r) and t-tests were used to assess association, Bland-Altman methods were used to evaluate systematic agreement between BFI and SAF fatigue scores, and Cronbach's alpha was used to measure internal consistency. Results There were 229 participants assigned both BFI and SAF fatigue items within the same survey. Among them, 51% (n=117) received the BFI item first and 49% (n=112) the SAF item first. No difference was seen between first and second fatigue scores (mean difference [first-second] = 0.00; 95%CI -0.18, 0.17). BFI and SAF fatigue scores were highly correlated (r=0.88, p<0.001) and showed 88.7% agreement in categorizing severe versus non-severe fatigue (score ≥ 7 versus < 7). Overall concordance in severity category was 73.4% (category [score range]: absent [0]; mild [1-3]; moderate [4-6]; severe [7-10]). Constructing the MPN-SAF TSS using the BFI and SAF fatigue components separately, the original and modified MPN-SAF TSS were nearly identical (r=0.997, p<0.001), and had equivalent internal consistency (both Cronbach's alpha=0.88). The Bland-Altman plot further indicates the 2 fatigue measures have high agreement with no evidence of directional bias and negligible overall bias (Figure 1: regression slope = -0.04, p=0.25; mean difference=0.22; 95%CI 0.05, 0.39). Within the COVID-19 survey (n=1217), the modified version of the MPN-SAF TSS was consistent with known correlates among disease characteristics (Emanuel R, et al. J Clin Oncol 2012). For example, more severe MPN-SAF TSS scores were highly correlated with low quality-of-life (n=1156, r = -0.50, p<0.001), and associated with those reporting spleen enlargement (n=301) versus not (n=617) (p<0.001; mean difference=7.7; 95%CI 5.4, 10.1). Conclusion The BFI and SAF fatigue items are highly consistent in raw score, severity category, and in contribution to the MPN-SAF TSS. There was no order effect seen in which fatigue item was asked first. The independent COVID-19 survey using the modified MPN-SAF TSS was validated and shows high internal consistent. In the ongoing development to capture the symptom experience, this analysis shows disease-related fatigue is equivalently measured using the SAF fatigue survey item in harmonization with the MFSAF v4. Disclosures Mesa: Bristol Myers Squibb: Research Funding; CTI BioPharma: Research Funding; Promedior: Research Funding; AbbVie: Research Funding; Samus Therapeutics: Research Funding; Genentech: Research Funding; Incyte: Research Funding; LaJolla Pharmaceutical Company: Consultancy; Novartis: Consultancy; Sierra Oncology: Consultancy.

The Myleloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) Derived Total Symptom Score (TSS): An International Trial of 1433 Patients with Myeloproliferative Neoplasms (MPNs),

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 3839-3839 ◽  
Author(s):  
Robyn Scherber ◽  
Amylou Dueck ◽  
Jean-Jacques Kiladjian ◽  
Stephanie Slot ◽  
Sonja Zweegman ◽  
...  
Keyword(s):  
Weight Loss ◽  
Construct Validity ◽  
Symptom Score ◽  
Internal Consistency ◽  
Convergent Validity ◽  
Bone Pain ◽  
Total Symptom Score ◽  
Early Satiety ◽  
Eortc Qlq C30 ◽  
Eortc Qlq

Abstract Abstract 3839 BACKGROUND: We have previously reported on the validation of the 18 item Myeloproliferative Neoplasm Assessment Form (MPN-SAF) (Blood 2011;118:401–408) given in conjunction with the 9 item Brief Fatigue Inventory (BFI) (Cancer 1999;85:1186–1196) to assess symptomatic burden in an international sample of MPN patients (pts), including validation in English, Italian, Swedish, German, French, Spanish, and Dutch. We desired to assess the utility of an average total symptom score (TSS) from the most pertinent and representative MPN symptoms for purposes of assessing the burden of symptoms in MPN pts, and subsequent tracking in response to therapy. METHODS: Data was collected among an international cohort of MPN pts and their physicians, including patient demographics and disease features and completion the BFI, MPN-SAF and the EORTC-QLQ-C30. Among pts who completed at least 5 of 10 specific items on the BFI and MPN-SAF, an average score was calculated as the TSS. TSS items included “worst” fatigue from the BFI and 9 items from the MPN-SAF including concentration, early satiety, inactivity, night sweats, itching, bone pain, abdominal discomfort, weight loss and fever. The TSS thus had a possible range of 0–10 with 10 representing the highest level of symptom severity. Data was then analyzed for internal consistency, and divergent, convergent validity, and construct validity. RESULTS: Patient Demographic and Disease Characteristics:1433 MPN pts were prospectively enrolled (Argentina 22, France 482, Germany 59, Italy 186, Netherlands 236, Puerto Rico 10, United Kingdom 57, United States 102, Spain 157, Sweden 114, Uruguay 8) including 594 ET, 538 PV and 293 MF pts (8 missing; MF: 61% Primary MF, 23% post-ET MF, 15% post-PV MF). 1408 pts completed at least 5 of the 10 items necessary to calculate a TSS. Pts were of characteristic age (mean 62, range 20–94) and gender (54% female) common to disease. TSS Burden of MPN Symptoms: Consistent with prior studies, the majority of pts (>50%) were symptomatic in each TSS item except for items associated with high disease severity, namely bone pain (48.6%), weight loss (30.6%) and fever (18.4%). Fatigue carried the highest symptom intensity (4.4, SD=2.8), followed by problems with concentration (2.5, SD=2.8) and early satiety (2.5, SD=2.7). Overall mean TSS was 2.1 (SD=1.6). Divergent Validity: TSS significantly differed among MPN disease subtypes (p<0.001) with means of 1.9 (SD=1.5), 2.2 (SD=1.6), and 2.5 (SD=1.7) for ET, PV, and MF pts, respectively. Statistically significant differences in TSS were also observed between pts with clinically deficient (>4, n=480) versus non-clinically deficient QOL (<4, n=894; mean 3.3 versus 1.5; p<0.001). When comparing to MD perceptions, TSS was significantly higher when MDs rated >2 of 6 common MPN-related symptoms as clinically significant (2.8, n=400) versus <2 symptoms (1.6, n=726; p<0.001). No significant trends were observed when comparing disease type by the presence of a current medical therapy. Convergent Validity: The TSS was strongly correlated with patient-reported QOL (r=0.59, p<0.001). Overall excellent correlations existed between the TSS and EORTC-QLQ-C30 functional subscales (all p<0.001 and r>0.50 except social functioning [r=0.48]). Additionally, excellent correlations were observed between the TSS and EORTC-QLQ-C30 fatigue and pain symptom scales (r>0.5, p<0.001). Internal Consistency and Construct Validity: The TSS had excellent internal consistency (Cronbach's alpha=0.83). Factor analysis identified a single underlying construct among the 10 TSS items (significant eigenvalues being >1). Factor loadings ranged from 0.43 for fever and weight loss to 0.71 for inactivity. The single factor suggests that the arithmetic mean of the 10 items is an appropriate global TSS score. CONCLUSION: The TSS demonstrated excellent psychometric properties. Overall, results of validity and internal consistency indicate that the TSS is a concise, valid, and accurate assessment of symptom burden among MPN pts. This new scoring will facilitate ease of implementation of the MPN-SAF into larger clinical trials and reduce ambiguity associated with interpreting response outcomes. Future analyses to investigate the impact of therapies on TSS are ongoing. Disclosures: No relevant conflicts of interest to declare.

Comparison of the Myleloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) Across Nine Linguistic Translations Among an International Sample of 1,851 Myeloproliferative Neoplasm (MPN) Patients.

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 2852-2852
Author(s):  
Amylou Constance Dueck ◽  
Robyn M. Emanuel ◽  
Holly Lynn Geyer ◽  
Jean-Jacques Kiladjian ◽  
Stephanie Slot ◽  
...  
Keyword(s):  
Board Of Directors ◽  
Factor Structure ◽  
Internal Consistency ◽  
Research Funding ◽  
Disease Type ◽  
Cronbach’S Alpha ◽  
Advisory Committees ◽  
Cronbach's Alpha ◽  
Language Groups ◽  
And Gender

Abstract Abstract 2852 Background: The 18-item Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF, Scherber et al Blood 2011) given in conjunction with the 9-item Brief Fatigue Inventory (BFI, Mendoza et al Cancer 1999) is a patient-completed questionnaire for assessing symptoms in persons with MPNs. The MPN-SAF has been translated and validated in 9 languages to date. The Total Symptom Score (TSS) is computed from 10 of the most pertinent MPN-SAF items to assess symptom burden in MPN patients and to evaluate response to therapy. Psychometric properties of the TSS have been previously reported (Emanuel et al Blood 2012). The purpose of this analysis is to compare MPN-SAF symptoms and psychometric properties of the TSS across 9 languages in an international sample. Methods: Data were collected in an international cohort of subjects with MPNs. Surveyed symptoms included fatigue, early satiety, abdominal pain and discomfort, inactivity, headaches, concentration, dizziness, extremity tingling, insomnia, sexual problems, mood changes, cough, night sweats, pruritus, bone pain and fever on a 0 (absent) to 10 (worst imaginable) scale. TSS was computed using the published scoring algorithm on a 0 (all symptoms absent) to 100 (all symptoms worst imaginable) scale. Demographic and disease-related data including disease type, gender, and age had to be present to be included in analysis. Demographics were compared across languages groups using ANOVA and chi-squared tests. Symptoms and TSS were compared across language groups using a general linear model adjusting for disease type, age, and gender with post-hoc Tukey pairwise comparisons. Internal consistency and factor structure of the TSS were investigated overall and within language groups using Cronbach's alpha and principal-axis factoring analysis. Results: Subject Demographics and Disease Type: 1,851 subjects with polycythemia vera (PV N=655), essential thrombocythemia (ET N=769) and myelofibrosis (MF N=427; 286 primary MF, 61 PV-MF, 80 ET-MF) were prospectively enrolled and administered the MPN-SAF and BFI in 1 of 9 languages: English [UK] 55, English [US] 102, Italian 186, Swedish 114, German 112, French 457, Spanish 192, Dutch 236, and Chinese 397. Age (median 61, range, 15–94) and gender (55% F) were typical. Disease type and age varied across language groups (both p <0.001). MPN-SAF Symptoms and TSS: Symptom frequencies ranged from 19% (fever) to 88% (fatigue) overall with mean severities ranging from 0.4 (SD=1.3, fever) to 4.3 (SD=2.3, fatigue). Fatigue had the highest mean severity among all symptoms within each language group. Overall, mean TSS was 21.5 (SD=16.7) with the Swedish (mean=18.1, SD=15.2) and Dutch (mean=27.6, SD=17.1) cohorts reporting the lowest and highest unadjusted TSS means, respectively. When comparing symptom items across languages (adjusting for disease type, age, and gender), concentration and sexual problems had the most statistically significant pairwise differences (11 and 10, respectively, out of a possible 36) followed by dizziness and overall quality of life (9 each, out of a possible 36). No statistically significant pairwise differences were observed for abdominal discomfort, headache, extremity tingling, or insomnia. For the TSS, the Dutch cohort appeared to statistically significantly differ (all p <0.05) with all other languages except the English cohorts. All other TSS pairwise comparisons were not statistically significant. TSS Internal Consistency and Factor Structure: The TSS had excellent internal consistency overall (Cronbach's alpha 0.83) as well as within language groups (Cronbach's alpha 0.81–0.86). Overall factor analysis identified a single underlying construct among the 10 TSS items. Factor loadings ranged from 0.41 for fever to 0.73 for inactivity. A single factor solution was appropriate for each language group with factor loadings ranging from 0.18 to 0.85. Conclusion: This analysis suggests that the available translations of the MPN-SAF are generally acceptable for use in a broad context. The TSS demonstrated acceptable internal consistency and similar factor structure across all language groups. Most symptom and TSS comparisons between languages were not statistically significant, but for the few which differed, further studies are needed to evaluate whether these variances are due to disease-related factors or due to linguistic or cultural influences present in the cohorts. Disclosures: Kiladjian: Novartis: Honoraria, Research Funding; Celgene: Research Funding; Shire: Honoraria. Griesshammer:Shire: Honoraria. Roy:Novartis, BMS: Speakers Bureau. Harrison:Novartis: Honoraria, Research Funding, Speakers Bureau; YM Bioscience: Consultancy, Honoraria; Sanofi Aventis: Honoraria; Shire: Honoraria, Research Funding. Passamonti:Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees; Celgene: Honoraria, Membership on an entity's Board of Directors or advisory committees; Sanofi: Honoraria, Membership on an entity's Board of Directors or advisory committees. Mesa:Incyte: Research Funding; Lilly: Research Funding; Sanofi: Research Funding; NS Pharma: Research Funding; YM Bioscience: Research Funding.

scholarly journals Myeloproliferative Neoplasm (MPN) Symptom Assessment Form Total Symptom Score: Prospective International Assessment of an Abbreviated Symptom Burden Scoring System Among Patients With MPNs

2012 ◽  
Vol 30 (33) ◽  
pp. 4098-4103 ◽  
Author(s):  
Robyn M. Emanuel ◽  
Amylou C. Dueck ◽  
Holly L. Geyer ◽  
Jean-Jacques Kiladjian ◽  
Stefanie Slot ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Symptom Score ◽  
Myeloproliferative Neoplasm ◽  
Symptom Burden ◽  
Symptom Assessment ◽  
Response To Therapy ◽  
Total Symptom Score ◽  
Life Questionnaire ◽  
Scoring Method

Purpose Myeloproliferative neoplasm (MPN) symptoms are troublesome to patients, and alleviation of this burden represents a paramount treatment objective in the development of MPN-directed therapies. We aimed to assess the utility of an abbreviated symptom score for the most pertinent and representative MPN symptoms for subsequent serial use in assessing response to therapy. Patients and Methods The Myeloproliferative Neoplasm Symptom Assessment Form total symptom score (MPN-SAF TSS) was calculated as the mean score for 10 items from two previously validated scoring systems. Questions focus on fatigue, concentration, early satiety, inactivity, night sweats, itching, bone pain, abdominal discomfort, weight loss, and fevers. Results MPN-SAF TSS was calculable for 1,408 of 1,433 patients with MPNs who had a mean score of 21.2 (standard deviation [SD], 16.3). MPN-SAF TSS results significantly differed among MPN disease subtypes (P < .001), with a mean of 18.7 (SD, 15.3), 21.8 (SD, 16.3), and 25.3 (SD, 17.2) for patients with essential thrombocythemia, polycythemia vera, and myelofibrosis, respectively. The MPN-SAF TSS strongly correlated with overall quality of life (QOL; r = 0.59; P < .001) and European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire C30 (EORTC QLQ-C30) functional scales (all P < .001 and absolute r ≥ 0.50 except social functioning r = 0.48). No significant trends were present when comparing therapy subgroups. The MPN-SAF TSS had excellent internal consistency (Cronbach's α = .83). Factor analysis identified a single underlying construct, indicating that the MPN-SAF TSS is an appropriate, unified scoring method. Conclusion The MPN-SAF TSS is a concise, valid, and accurate assessment of MPN symptom burden with demonstrated clinical utility in the largest prospective MPN symptom study to date. This new prospective scoring method may be used to assess MPN symptom burden in both clinical practice and trial settings.

scholarly journals Validation of a Modified Version of the Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score

2021 ◽  
Vol 10 (5) ◽  
pp. 207-211
Author(s):  
Blake T. Langlais ◽  
Gina L. Mazza ◽  
Heidi E. Kosiorek ◽  
Jeanne Palmer ◽  
Ruben Mesa ◽  
...  
Keyword(s):  
Symptom Score ◽  
Myeloproliferative Neoplasm ◽  
Symptom Assessment ◽  
Total Symptom Score

scholarly journals HOW DO LEARNING DISORDERS IMPACT CLINICAL MEASURES FOLLOWING CONCUSSION?

2020 ◽  
Vol 8 (4_suppl3) ◽  
pp. 2325967120S0018
Author(s):  
Aaron J. Zynda ◽  
Mathew A. Stokes ◽  
Jane S. Chung ◽  
C. Munro Cullum ◽  
Shane M. Miller
Keyword(s):  
Symptom Score ◽  
Learning Disorders ◽  
Total Symptom Score ◽  
Adhd Group ◽  
Motor Speed ◽  
The North ◽  
History Of ◽  
Visual Motor ◽  
Clinical Measures ◽  
The Impact

Background: There is limited evidence examining the impact of learning disorders on testing and screening scores used in evaluation following concussion in adolescents. Purpose: To examine differences in clinical measures between adolescents with a history of dyslexia or ADD/ADHD and those without a history of learning disorder (LD) following concussion. Methods: Data were collected from participants enrolled in the North Texas Concussion Network Prospective Registry (ConTex). Participants ages 10-18 who had been diagnosed with a concussion sustained within 30 days of enrollment were included. Participants were separated into three groups based on self-reported prior diagnosis: dyslexia, ADD/ADHD, and no history of LD. Clinical measures from initial presentation were examined, including ImPACT®, King-Devick (KD), SCAT-5 symptom log, Patient Health Questionnaire (PHQ-8), and Generalized Anxiety Disorder (GAD-7) scale. Independent t-test analysis was performed to compare scores between groups. Results: A total of 993 participants were included; 68 with dyslexia, 141 with ADD/ADHD, and 784 with no history of LD. There was no difference in age, sex, time since injury, or history of concussion between the dyslexia group and no LD group. In the ADD/ADHD group, there were significantly more male participants (64.5% and 50.3% respectively, p=0.002). Participants with a history of dyslexia had a significant increase in KD time (63.7 sec vs 56.5 sec, p=0.019). Additionally, ImPACT® testing showed a decrease in visual motor speed (28.87 vs 32.99, p= 0.010). Total symptom score was higher in this group as well (36.22 vs 28.27, p=0.013). In those with a history of ADD/ADHD, multiple domains were found to be significantly different on ImPACT® testing including visual motor speed (30.05), reaction time (0.75), and cognitive efficiency (0.23) when compared to those with no LD (32.99, 0.71, and 0.27 respectively, p=0.004, 0.047, 0.027). KD time was also significantly higher in this group (62.1 sec vs 56.5 sec, p=0.008), as was the total symptom score (32.99 vs 28.27, p=0.043). PHQ-8 and GAD-7 were both significantly higher in the group with ADD/ADHD (5.79 and 5.06 respectively, p=0.001) than those with no LD (4.32 and 3.56, p=0.001). Conclusion: Differences were seen in participants with a history of dyslexia and ADD/ADHD on clinical concussion measures, including ImPACT® and KD testing, SCAT-5 symptom log, and screenings for depression and anxiety. A better understanding of the unique profiles seen in these patients will aid providers in their evaluation and assist as they counsel families regarding their child’s injury.

scholarly journals Reliability and validity of the functioning assessment short test for older adults with bipolar disorder (FAST-O)

2020 ◽  
Vol 8 (1) ◽  
Author(s):  
Melis Orhan ◽  
Nicole Korten ◽  
Ralph Kupka ◽  
Patricia van Oppen ◽  
Max Stek ◽  
...  
Keyword(s):  
Older Adults ◽  
Bipolar Disorder ◽  
Internal Structure ◽  
Internal Consistency ◽  
Concurrent Validity ◽  
Cronbach’S Alpha ◽  
Occupational Functioning ◽  
Cronbach's Alpha ◽  
Short Test ◽  
Functioning Assessment

Abstract Background Many frequently used instruments fail to assess psychosocial functioning in patients with bipolar disorder. The Functioning Assessment Short Test (FAST) was developed in order to tackle this problem and to assess the main functioning problems experienced by patients with bipolar disorder. However, the original FAST is not fully applicable in older adults due to the domain of occupational functioning. The aim of our study was to validate an adapted version for Older adults (FAST-O) in a group of older adults with bipolar disorder (OABD). Methods 88 patients aged 50 years and over diagnosed with bipolar disorder were included. We adapted the items in the area of “work-related functioning” of the FAST into items assessing “societal functioning”. Several measurements were conducted in order to analyse the psychometric qualities of the FAST-O (confirmatory factor analysis for internal structure, Cronbach’s alpha for internal consistency, Spearman’s rho for concurrent validity, Mann–Whitney U test for discriminant validity). Results Mean age in the study sample was 65.3 (SD = 7.5) and 57.3% was female. The internal structure was most similar to the internal structure of the original FAST. The internal consistency was excellent (Cronbach’s alpha = .93). The concurrent validity when correlated with the Social and Occupational Functioning Assessment Scale was low, but significant. The FAST-O was also able to distinguish between euthymic and symptomatic OABD patients. Conclusions The FAST-O has strong psychometric qualities. Based on our results, we can conclude that the FAST-O is a short, efficient solution in order to replace global rating scales or extensive test batteries in order to assess daily functioning of older psychiatric patients in a valid and reliable manner.

scholarly journals Development and content validity of the Barth Syndrome Symptom Assessment (BTHS-SA) for adolescents and adults

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Chad Gwaltney ◽  
Jonathan Stokes ◽  
Anthony Aiudi ◽  
Iyar Mazar ◽  
Sarah Ollis ◽  
...  
Keyword(s):  
Clinical Studies ◽  
Genetic Disorder ◽  
Signs And Symptoms ◽  
Symptom Assessment ◽  
Barth Syndrome ◽  
Therapeutic Area ◽  
Symptom Experience ◽  
Patient Reported ◽  
Adolescents And Adults ◽  
Syndrome Symptom

Abstract Background Barth Syndrome (BTHS) is a rare genetic disorder that presents as a complex of debilitating symptoms and reduced life expectancy. Well-developed, BTHS-specific assessments measuring primary signs and symptoms of BTHS are not currently available, making it difficult to evaluate treatment effects in BTHS clinical studies. The objective of this research was to develop symptom-focused patient-reported outcome (PRO) measures for use in clinical studies with adolescents and adults with BTHS. Methods Concept elicitation interviews (CEIs) with pediatric (n = 18, age < 16 years) and adult (n = 15, age ≥ 16 years) individuals with BTHS and/or their caregivers were conducted to identify signs and symptoms relevant to BTHS and important to individuals with the condition. Based on CEI results, questionnaire construction activities were conducted to create unique adolescent and adult versions of the Barth Syndrome-Symptom Assessment (BTHS-SA). The questionnaires were evaluated in cognitive debriefing interviews (CDIs) with adolescents (n = 12; age 12- < 16 years) and adults (n = 12; age ≥ 16 years) with BTHS to assess relevance and readability of the tools. Results During the CEIs, a total of 48 and 40 signs and symptoms were reported by the pediatric and adult groups, respectively; 31 were reported by both age groups. Fatigue/tiredness and muscle weakness were the symptoms most frequently reported by both pediatric and adult patients with BTHS as important to improve with an effective treatment. The CEI results informed construction of a nine-item version of the BTHS-SA for adolescents and an eight-item version for adults. Developed for daily administration, each version asks respondents to rate symptom severity “at its worst” over the 24 h prior to administration. CDIs with both adolescents and adults with BTHS demonstrated that each BTHS-SA version was reflective of the disease experience and that respondents could interpret the questionnaire as intended and provide responses that accurately reflected their symptom experience. Conclusions The BTHS-SA adolescent and adult versions are content-valid PRO measures that can be used to evaluate severity of disease-specific symptoms in future clinical trials. Given the lack of available and well-developed assessments in this underserved therapeutic area, these tools fulfill a need for clinical researchers developing treatments for individuals with BTHS.

scholarly journals The development of a contextually appropriate measure of psychological distress in Sierra Leone

2021 ◽  
Vol 9 (1) ◽  
Author(s):  
Rebecca Horn ◽  
Kanykey Jailobaeva ◽  
Stella Arakelyan ◽  
Alastair Ager
Keyword(s):  
Mental Health ◽  
Factor Analysis ◽  
Psychological Distress ◽  
Sierra Leone ◽  
Exploratory Factor Analysis ◽  
Internal Consistency ◽  
Phase 1 ◽  
Cronbach’S Alpha ◽  
Cronbach's Alpha ◽  
Distress Scale

Abstract Background Studies of psychological distress in Sierra Leone have typically used measures which were developed for use in other contexts, and which often have not been adapted or validated for use in Sierra Leone. This has resulted in a lack of reliable information about the patterns of psychological distress within the population, which is a barrier to the development of effective and appropriate mental health services. The aim of the study was to develop a locally-appropriate measure of psychological distress for Sierra Leone. Methods The new measure consists of two instruments: the Sierra Leone Psychological Distress Scale (SLPDS) and a gendered measure of ability to carry out daily tasks—a Function scale—as an indication of the severity of distress. A three-phase mixed methods exploratory sequential study was conducted. Phase 1 was item generation and testing, leading to the development of a set of potential items for both instruments. Phase 2 was a small pilot study (N = 202) leading to the selection of the final set of items for both measures. Phase 3 was a validation phase where the SLPDS and the Function scale were administered with a larger sample of 904 respondents. Item analysis was used to assess the internal consistency of the scales, and Exploratory Factor Analysis to explore the properties of the SLPDS. Results Exploratory factor analysis using the principal axis factoring with an oblique rotation identified a three-factor structure for the 18-item SLPDS. Internal consistency for the SLPDS (Cronbach’s alpha = 0.89) and three subscales was good (Cronbach’s alpha > 0.73). The internal reliability of the male and female versions of the Function scale was also found to be acceptable (Cronbach’s alpha = 0.90 for the female scale and 0.79 for the male scale). Conclusions Together the SLPD and Function scales provide a locally-validated tool which will enable government bodies and local and international non-governmental organisations in Sierra Leone to assess mental health and psychosocial needs. This will support both effective service provision and the evaluation of initiatives designed to improve mental health and psychosocial wellbeing.

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