The Pediatric Toronto Extremity Salvage Score (pTESS): Validation of a Self-reported Functional Outcomes Tool for Children with Extremity Tumors.

BACKGROUND: The physical function of children with sarcoma after surgery has not been studied explicitly. This paucity of research is partly because of the lack of a sufficiently sensitive pediatric functional measure. The goal of this study was to establish and validate a standardized measure of physical function in pediatric patients with extremity tumors. QUESTIONS/PURPOSES: (1) What is the best format and content for new upper- and lower-extremity measures of physical function in the pediatric population? (2) Do the new measures exhibit floor and/or ceiling effects, internal consistency, and test-retest reliability? (3) Are the new measures valid? METHODS: In Phase 1, interviews with 17 consecutive children and adolescents with bone tumors were conducted to modify the format and content of draft versions of the pediatric Toronto Extremity Salvage Score (pTESS). In Phase 2, the pTESS was formally translated into French. In Phase 3, 122 participants between 7 and 17.9 years old with malignant or benign-aggressive bone tumors completed the limb-specific measure on two occasions. Older adolescents also completed the adult TESS. Floor and ceiling effects, internal consistency, test-retest reliability, and validity were evaluated. RESULTS: Feedback from interviews resulted in the removal, addition, and modification of draft items, and the pTESS-Leg and pTESS-Arm questionnaires were finalized. Both versions exhibited no floor or ceiling effects and high internal consistency (α > 0.92). The test-retest reliability was excellent for the pTESS-Leg (intraclass correlation coefficient [ICC] = 0.94; 95% CI, 0.90-0.97) and good for the pTESS-Arm (ICC = 0.86; 95% CI, 0.61-0.96). Known-group validity (ability to discriminate between groups) was demonstrated by lower mean pTESS-Leg scores for participants using gait aids or braces (mean = 68; SD = 21) than for those who did not (mean = 87; SD = 11; p < 0.001). There was no significant difference between pTESS arm scores among respondents using a brace (n = 5; mean = 73; SD = 11) and those without (n = 22; mean = 83; SD = 19; p = 0.13). To evaluate construct validity, we tested a priori hypotheses. The duration since chemotherapy correlated moderately with higher pTESS-Leg scores (r = 0.4; p < 0.001) but not with pTESS-Arm scores (r = 0.1; p = 0.80), and the duration since tumor resection correlated moderately with higher pTESS-Leg scores (r = 0.4; p < 0.001) but not pTESS-Arm scores (r = 0.2; p = 0.4). Higher VAS scores (that is, it was harder to do things) antecorrelated with both pTESS versions (pTESS-Leg: r = -0.7; p < 0.001; pTESS-Arm: r = -0.8; p < 0.001). To assess criterion validity, we compared the pTESS with the current "gold standard" (adult TESS). Among adolescents, strong correlations were observed between the TESS and pTESS-Leg (r = 0.97, p < 0.001) and pTESS-Arm (r = 0.9, p = 0.007). CONCLUSIONS: Both pTESS versions exhibited no floor or ceiling effects and had high internal consistency. The pTESS-Leg demonstrated excellent reliability and validity, and the pTESS-Arm demonstrated good reliability and reasonable validity. The pTESS is recommended for cross-sectional evaluation of self-reported physical function in pediatric patients with bone tumors. LEVEL OF EVIDENCE: Level II, outcome measurement development.

EXERCISE in pediatric autologous stem cell transplant patients: a randomized controlled trial protocol.

BACKGROUND: Hematopoietic stem cell transplantation is an intensive therapy used to improve survivorship and cure various oncologic diseases. However, this therapy is associated with high mortality rates and numerous negative side-effects. The recovery of the immune system is a special concern and plays a key role in the success of this treatment. In healthy populations it is known that exercise plays an important role in immune system regulation, but little is known about the role of exercise in the hematological and immunological recovery of children undergoing hematopoietic stem cell transplant. The primary objective of this randomized-controlled trial (RCT) is to study the effect of an exercise program (in- and outpatient) on immune cell recovery in patients undergoing an autologous stem cell transplantation. The secondary objective is to determine if an exercise intervention diminishes the usual deterioration in quality of life, physical fitness, and the acquisition of a sedentary lifestyle. METHODS: This RCT has received approval from The Conjoint Health Research Ethics Board (CHREB) of the University of Calgary (Ethics ID # E-24476). Twenty-four participants treated for a malignancy with autologous stem cell transplant (5 to 18 years) in the Alberta Children's Hospital will be randomly assigned to an exercise or control group. The exercise group will participate in a two-phase exercise intervention (in- and outpatient) from hospitalization until 10 weeks after discharge. The exercise program includes strength, flexibility and aerobic exercise. During the inpatient phase this program will be performed 5 times/week and will be supervised. The outpatient phase will combine a supervised session with two home-based exercise sessions with the use of the Wii device. The control group will follow the standard protocol without any specific exercise program. A range of outcomes, including quantitative and functional recovery of immune system, cytokine levels in serum, natural killer (NK) cells and their subset recovery and function, and gene expression of activating and inhibitory NK cell receptors, body composition, nutrition, quality of life, fatigue, health-related fitness assessment and physical activity levels will be examined, providing the most comprehensive assessment to date. DISCUSSION: We expect to find improvements in immunological recovery and quality of life, and decreased acquisition of sedentary behavior and fitness deconditioning. The comprehensive outcomes generated in this RCT will provide preliminary data to conduct a multisite study that will generate stronger outcomes. TRIAL REGISTRATION: Gov identification # NCT01666015.