Prospective development of a patient-reported outcomes instrument for desmoid tumors or aggressive fibromatosis.

BACKGROUND: Desmoid tumors (or aggressive fibromatosis) are locally infiltrative connective-tissue tumors that can arise in any anatomic location; they can be asymptomatic, or they can result in pain, deformity, swelling, and loss of mobility and/or threaten visceral organs with bowel perforation, hydronephrosis, neurovascular damage, and other complications. Existing clinical trial endpoints such as the Response Evaluation Criteria in Solid Tumors (version 1.1) and progression-free survival are inadequate in capturing treatment efficacy. This study was designed to develop a novel clinical trial endpoint by capturing patient-reported outcomes (PROs). METHODS: Following best practices in qualitative methodology, this study used concept elicitation (CE) interviews to explore desmoid patients' perspectives on key disease-related symptoms and impacts. Qualitative analysis was performed to determine the relative frequency and disturbance of symptoms and impacts as well as other characteristics of these concepts. A draft PRO scale was then developed and tested with cognitive interviewing. Information from the interviews was subsequently incorporated into the refined PRO scale. RESULTS: CE interviews with desmoid patients (n = 31) helped to identify salient concepts and led to a draft scale that included symptom and impact scales. Cognitive interviews were completed with additional patients (n = 15) across 3 phases. Patient input was used to refine instructions, revise and/or remove items, and modify the response scale. This resulted in an 11-item symptom scale and a 17-item impact scale. CONCLUSIONS: This is the first disease-specific PRO instrument developed for desmoid tumors. The instrument is available as an exploratory endpoint in clinical trials. This study highlights the feasibility and challenges of developing PRO instruments for rare diseases.

Impact of palliative urinary diversion by percutaneous nephrostomy drainage and ureteral stenting among patients with advanced cervical cancer and obstructive uropathy: a prospective cohort.

AIM: Obstructive uropathy is a recognized complication in advanced cervical cancer. Urinary diversion is commonly used to bypass the obstruction and improve renal function. The degree of survival benefit that diversion offers is not well established and its impact on quality of life (QoL) is uncertain. This study considered these factors in order to inform treatment decisions. METHODS: This study examined a prospective cohort of patients with advanced cervical cancer and obstructive uropathy in Manila, Philippines. Age, cancer treatment status, comorbidities, serum creatinine level, degree of obstructive uropathy and QoL were recorded at baseline. Patients with creatinine values >150 µmol/L, or who were being considered for radiotherapy or nephrotoxic chemotherapy or manifesting uncontrolled or recurrent uropathy-related urinary tract infection, were offered diversion. Follow-up data collection was at 3, 6, 9 and 12 months from cohort entry. RESULTS: Of the 230 patients invited, 205 patients joined the cohort. Complete data were available for 198, of whom 93 underwent diversion, 56 required diversion but elected not to receive it, and 49 did not require it. Although survival at 12 months among those who underwent diversion was no greater than among those who required but elected not to receive the procedure, diversion was associated with significantly improved chance of survival in the shorter term. There was no significant difference in the QoL between the groups throughout the study. CONCLUSION: With no evidence of an impact on QoL, the decision to offer diversionary surgery might be based solely on a survival benefit, which is modest but potentially important to patients.

Prospective evaluation of health related quality of life for pediatric patients with ureteropelvic junction obstruction.

PURPOSE: Clinical practices are increasingly proposing health related quality of life measures for informed treatment decisions. Dismembered pyeloplasty is an accepted standard therapy for ureteropelvic junction obstruction. This study evaluates health related quality of life in patients with ureteropelvic junction obstruction undergoing pyeloplasty. Patients have perceived it helpful in communicating health care needs to physicians. We believe this is the first study to use child self and parent reports prospectively in ureteropelvic junction obstruction. The goals of the study were to document baseline preoperative health related quality of life assessments, differences between parent and child assessments at given intervals, differences in preoperative and postoperative assessments, and overall clinical outcomes. MATERIALS AND METHODS: Patients younger than 18 years (mean 9.1 years) with ureteropelvic junction obstruction were included in the study. Demographic survey and validated health related quality of life questionnaire (Pediatric Quality of Life Inventory 4.0) were used preoperatively and postoperatively. The questionnaire documented subjective health related quality of life (physical, social, emotional and school functioning, and psychosocial health). Clinical outcomes were generated following the office visit. Questionnaire subscales were scored with algorithms provided. Paired t test evaluated differences in parent and child scores of less than 0.05 were statistically significant. Tests were 2-tailed. RESULTS: Response rate was 100%. Preoperatively emotional functioning (81.8) and psychosocial health (80.9) child scores were significantly higher than parent scores (70.7 and 73.9, respectively). Overall child score of the study population (80.9) was similar to that of healthy children (85). However, parent scores of physical functioning (78.3), psychosocial health (73.9), emotional functioning (70.7) and school functioning (65.5) were significantly lower than the general population. At postoperative week 6 child emotional functioning (91.7) and physical functioning (90.3) showed significant improvement (p <0.05). Parent scores of physical functioning (88.4), psychosocial health (82.2) and emotional functioning (80.8) were also significantly higher than preoperative scores. Longer followup demonstrated that child scores of physical functioning (96.9), psychosocial health (96.5), emotional functioning (95.4) and social functioning (97.1) were significantly higher than preoperatively. Postoperatively parents reported significantly higher health related quality of life scores compared to preoperative scores. There was no significant difference at 6 months between parent and child scores. Clinically all patients did well following pyeloplasty. CONCLUSIONS: Preoperatively children recorded higher health related quality of life than parents/guardians. At postoperative week 6 children and parents recorded higher health related quality of life compared to preoperative scores. At 6 months overall child health related quality of life was significantly higher than preoperative reports, and no significant difference was seen between parent and child scores. Health related quality of life evaluations enabled us to monitor patient recovery and progress postoperatively. Prospective evaluations at regular intervals helped us to document improvement in overall quality of life in these children.

[Urogenital symptoms and possibilities for urological intervention in the palliative care situation]. / Urogenitale Symptome und urologische Interventionsmöglichkeiten in der palliativen Situation.

During the course of malignancies of nearly all tumor entities the urogenital organs are frequently influenced. The resulting disorders are subsumed under the term"urogenital symptoms". Especially with the goal of improving quality of life these symptoms have to be treated with respect. In addition further therapeutic measures, e.g. the application of a palliative chemotherapy, makes an unobstructed urinary excretion necessary. This article gives an overview of the indications for treating urogenital symptoms and contrasts different therapy concepts.

Antenatal minimal hydronephrosis: is its follow-up an unnecessary cause of concern?

The aim of this study was to determine if follow-up of antenatally diagnosed minimal hydronephrosis (anteroposterior renal pelvis diameter <10 mm) is justified or if it is an unnecessary cause of concern for the parents involved. A case-control study, with subjects and controls selected from the Wessex Antenatally Detected Anomalies Register was performed. Information regarding antenatal and postnatal follow-up, renal tract morbidity and degree of concern was obtained from a parental questionnaire. 70 of the 115 subjects contacted (60.9 per cent) and 52 of the 81 controls (64.2 per cent) returned the questionnaire. 65 of the 70 subjects (92.9 per cent) were rescanned postnatally when 28 cases (43.1 per cent) had resolved. Coexisting ureteric and/or calyceal dilatation was evident postnatally in 12 cases and this group was significantly more likely to have underlying pathology than the group with isolated renal pelvis dilatation. Subjects were significantly more likely than controls to have a UTI. The degree of concern was significantly greater in the subject group and subject parents thought about the result significantly more often than controls. From our results we concluded that the follow-up of minimal hydronephrosis can be modified. There is no need for repeated antenatal scanning, a change that could reduce the level of parental anxiety. Postnatal follow-up is required in all patients to exclude an underlying uropathy but again this can be modified, with the majority of patients requiring only an ultrasound scan. This reduced intensity of investigation accompanied with careful explanation to the parents should help to minimize their concerns.

Reduction of hyponatremia in a schizophrenic with polydipsia-hyponatremia syndrome by surgical intervention.

In a chronic schizophrenic with polydipsia-hyponatremia syndrome, we observed physiological data consecutively before and after a successful LAPIDES vesicostomy for his bladder retention. Although his polydipsia was unchanged, frequency of hyponatremia was significantly reduced after the operation. We found that bladder retention might be one of the factors relevant to the prediction of hyponatremia from diurnal weight gain.