Psychometric properties of the FACT-M questionnaire in patients with Merkel cell carcinoma

Background

No validated disease-specific questionnaires exist to capture health-related quality of life (HRQoL) in patients with Merkel cell carcinoma (MCC). The Functional Assessment of Cancer Therapy – Melanoma (FACT-M) is validated in patients with melanoma, which shares many similarities with MCC. This paper reports the psychometric properties of the FACT-M in the metastatic MCC population.

Methods

Data were collected as part of a single-arm, open-label, multicenter trial involving patients with metastatic MCC who had failed at least one previous line of chemotherapy. FACT-M and EQ-5D were administered at baseline, Week 7, Week 13, and Week 25. An optional interview was administered at the same time points. MCC-specific FACT-M scores were derived following a combined quantitative and qualitative approach. Reliability and construct validity of original and additional MCC-specific FACT-M scores were assessed at baseline. Capacity to detect change in tumor size was assessed from baseline to Week 7. Minimally important differences (MIDs) were computed using distribution and anchor-based methods.

Results

Baseline assessments were available in 70 patients (mean age: 70 years; 74.3% male); 19 patients were interviewed at baseline. Additional MCC-specific scores were as follows: Physical Function score (six items), Psychological Impact score (six items), and MCC summary score (12 items). FACT-M original and additional MCC-specific scores both demonstrated acceptable psychometric properties: high reliability (Cronbach’s alpha: 0.81–0.96), good convergent validity (correlations above 0.4 observed for 88% of items of the Melanoma surgery scale, 75% of items of the Melanoma scale, and 100% of items of the other FACT-M domains). Some evidence of floor/ceiling effects and poor discriminant ability was found. Higher scores (better HRQoL) on all FACT-M domains were observed in patients with better functioning (assessed by ECOG performance score), supporting clinical validity. Despite the small sample for responsiveness analysis (n =?37), the majority of FACT-M scores showed sensitivity to changes in tumor size at Week 7 with small to moderate effect sizes. MIDs were consistent with previously reported values in the literature for FACT-M domains.

Conclusions

FACT-M is suitable to capture HRQoL in patients with metastatic MCC, thus making it a potential candidate for assessing HRQoL in MCC trials.

Trial registration

This study is a post-hoc analysis conducted on data collected in Part A of the JAVELIN Merkel 200 trial. This trial was registered on 2 June 2014 with ClinicalTrials.gov as NCT02155647.

     

La fonctionnalité alimentaire : illusion aujourd’hui, réalité demain

Foods have always been considered by man as a means of ensuring his physiological needs, allowing the growth, development and preservation of the body and its tissues (nutritional value of food). To this can be added the feelings of satisfaction and well-being that food gives to its consumer, thus constituting an element that is fundamental for our physiological and mental balance (the sensorial value of food).Nevertheless, recent scientific studies have shown that over and above the ensuring of nutritional needs, eating habits can also adjust certain functions of the human organism and thus play a beneficial or harmful role on one's health (the functional value of food).The whole concept of nutrition has been enriched by the notion that eating is not only a survival reflex (satisfaction derived through eating and the avoidance of harmful effects due to eating deficiencies or excesses): eating aims to improve one's health and well-being and to reduce the risk of developing various pathologies. These new data open interesting new horizons in today's context where health is increasingly expensive and people are increasingly concerned to improve their quality of live.     

External validation of a prognostic nomogram for overall survival in women with uterine leiomyosarcoma

BACKGROUND:

There is no validated system to identify prognostically distinct cohorts of women with uterine leiomyosarcoma (ULMS). By using an independent, pooled, multi‐institutional, international patient cohort, the authors validated a recently proposed ULMS nomogram.

METHODS:

The ULMS nomogram incorporated 7 clinical characteristics (age, tumor size, tumor grade, cervical involvement, locoregional metastases, distant metastases, and mitotic index (per 10 high‐power fields) to predict overall survival (OS) after primary surgery. Independent cohorts from 2 sarcoma centers were included. Eligible women, at minimum, underwent a hysterectomy for primary, locally advanced, or metastatic ULMS and received part of their care at 1 of the centers between 1994 and 2010.

RESULTS:

In total, 187 women with ULMS were identified who met the above criteria described above (median age, 51 years; median tumor size, 9 cm; median mitotic index, 20 per 10 high‐power fields). Tumors generally were high grade (88%), FIGO stage I or II (61%) without cervical involvement (93%) and without locoregional metastases (77%) or distant metastases (83%). The median OS and the 5‐year OS rate were 4.5 years (95% confidence interval, 3.2‐5.3 years) and 46%, respectively; and 65 women (35%) remained alive at last follow‐up. The nomogram concordance index was 0.67(standard error, 0.02), which was as high as the concordance index from the initial cohort used for nomogram development. The concordance between actual OS and nomogram predictions suggests excellent calibration because predictions were within 1% of actual 5‐year OS rates for patients with a predicted 5‐year OS of less than 0.68.

CONCLUSIONS:

The ULMS nomogram was externally validated using independent cohorts. These findings support the international use of the ULMS nomogram prognostic of OS in ULMS. Cancer 2013. © 2012 American Cancer Society.     

HtrA1 in human urothelial bladder cancer: A secreted protein and a potential novel biomarker

Our aim was to analyze the expression of the serine protease HtrA1 in human bladder tissue and urine in order to point out its possible association with the presence of urothelial bladder cancer. Bladder tissue and urine specimens from cancer patients with different tumor grades and stages (n = 68) and from individuals with cystitis (n = 16) were collected along with biopsy specimens and urine from healthy individuals (n = 68). For the first time, we demonstrated by immunohistochemistry that HtrA1 protein is produced by bladder urothelium in both physiological and inflammatory conditions, whereas it is not detectable in urothelial cancer cells regardless of tumor grade and stage. A different HtrA1 expression between normal‐looking and neoplastic bladder tissue, despite similar HtrA1 mRNA levels, was also found by western blotting, which disclosed the presence of two forms of HtrA1, a native form of ～50 kDa and an autocatalytic form of ～38 kDa. Our investigations documented the presence of the two forms of HtrA1 also in urine. The ～38 kDa form was significantly down‐regulated in neoplastic tissue, whereas significantly higher amounts of both HtrA1 forms were found in urine from cancer patients compared with both healthy subjects and patients with cystitis. Our findings suggest that HtrA1 is a downexpressed molecule since an early stage of bladder urothelial carcinoma development and that urinary HtrA1 protein may be considered, if successfully validated, as an early and highly sensitive and specific biomarker for this neoplasia (the sensitivity and specificity of HtrA1 are 92.65% and 95.59%, respectively).     

The validity of simple methods to detect poor nutritional status in paediatric oncology patients

Poor nutritional status is a major concern for children being treated for an oncology condition. It is vital for optimal prognosis that nutritional concerns are recognised and treated promptly. The majority of oncology treatment centres only use simple methods to screen for poor nutritional status; however, it is unknown whether these simple methods accurately recognise poor nutritional status. We aimed to determine whether commonly used simple nutritional assessment variables could accurately identify poor nutritional status in children being treated for oncological conditions. This cross-sectional study measured height, weight, mid-upper arm circumference, triceps skinfold, albumin levels and body cell mass in forty children being treated for an oncological condition. To determine whether commonly used nutritional variables were suitable determinants, the variables were compared against the reference measure of body cell mass index (BCMI) Z-scores. Using the BCMI Z-score cut-off of - 1.65, 48 % of the study population were considered poorly nourished. Correlational analysis showed that there was no significant biological relationship between the BCMI Z-score and the simple parameters. When divided into two groups based on the nutritional status indicated by the BCMI Z-score, the independent t tests between the well-nourished and malnourished groups demonstrated that there was a significant difference in the BMI Z-score (P = 0.01) between the groups. No simple nutritional measures were found to accurately identify poor nutritional status in children being treated for oncological conditions.