Kidney Stone, Nephrolithiasis
Conditions
Keywords
kidney stones, kidney, nephrolithiasis, Comparative Effectiveness, Patient Reported Outcomes
Brief summary
The aims of the Observational Approach versus Surgical Intervention for Asymptomatic Stone (OASIS) trial are: 1) to determine whether observation compared to upfront surgery results in less healthcare-related life disruption due to kidney stones among children and adults with asymptomatic kidney stones; 2) to identify the groups benefiting the most from each strategy; and 3) to determine the preferences and values informing the choice between observation and upfront surgery.
Detailed description
A critical barrier to improving the health of the nearly 40 million Americans with kidney stone disease is the weak evidence base supporting the management of individuals with asymptomatic kidney stones. The proposed Observational Approach versus Surgical Intervention for Asymptomatic Stone (OASIS) randomized clinical trial answers the research question of whether observation or upfront surgery results in less life disruption from asymptomatic kidney stones for children and adults. The management options for patients with asymptomatic stones are 1) observation or 2) upfront surgical treatment, both of which are considered safe, part of usual care, widely accessible, and covered by insurance. OASIS responds to the charge for well-designed Randomized Controlled Trials (RCT) from the Agency for Healthcare Research and Quality (AHRQ) systematic review on kidney stone disease management and ensures patient centeredness by examining outcomes that patients have identified as meaningful. Intervention Randomization to either: 1. Observation is the surveillance of asymptomatic stones with periodic CT, ultrasound, or x-ray, and clinic visits. 2. Upfront surgery is the planned removal of asymptomatic stones and includes ureteroscopy (URS) and shockwave lithotripsy (SWL), which are routinely used in standard clinical care. Observational Cohort (Non-Randomized Cohort): Up to 300 eligible subjects who are willing to consent to data collection but refuse randomization will be enrolled and followed for two years. Study Design OASIS is a pragmatic randomized controlled superiority trial conducted at approximately 30 sites that participate in Patient Centered Outcomes Research Network (PCORnet). The trial will include an additional observational cohort for eligible subjects who decline randomization.
Interventions
PROCEDURESurgery
Upfront surgery is the planned removal of asymptomatic stones and includes ureteroscopy and shockwave lithotripsy, which are routinely used in standard clinical care.
OTHERObservation
Observation is the surveillance of asymptomatic stones with periodic CT, ultrasound, or x-ray, and clinic visits.
Sponsors
Children's Hospital of Philadelphia
Patient-Centered Outcomes Research Institute
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Outcomes Assessor)
Masking description
Allocation concealment will be achieved by having the randomization performed centrally at the Data Coordinating Center (DCC) in REDCap, with randomly permuted blocks of 4, 6, and 8 to minimize the risk of allocation bias. Additionally, we will require participant details to be entered into the system before the randomly allocated treatment is revealed. Blinding will not be possible for participants or investigators providing the treatment (observation or upfront surgery). Members of the Outcome Adjudication Committee will be blinded to group assignment. The principal investigators will not have access to participant level information.
Eligibility
Sex/Gender
ALL
Age
6 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Individuals age 6 or older 2. Diagnosis of kidney stone disease (nephrolithiasis) 3. Asymptomatic, non-obstructive kidney stones measuring 4 to 10 mm (inclusive) * Asymptomatic is defined as the absence of abdominal pain, flank pain, gross hematuria, and nausea and/or vomiting in the last 3 months that in the judgement of the site PI could be attributable to kidney stones. * Non-obstructive is defined as a stone in a renal calyx or stones in multiple renal calyces on CT, ultrasound, and/or x-ray obtained within 6 months of screening. 4. Informed consent and if appropriate, child assent. 5. Willingness to be randomized to surgery or observation Note: subjects otherwise eligible who decline randomization may be enrolled in a parallel observational cohort. 6. Fluent in English or Spanish 7. Willingness to be audio- and/or video- recorded during interviews
Exclusion criteria
1. Untreated hydronephrosis or caliectasis 2. Pain (e.g., renal colic, abdominal pain) or gross hematuria attributed to kidney stones. 3. Recurrent symptomatic urinary tract infections (≥3 in 12 months or ≥2 in 6 months). A symptomatic urinary tract infection is defined as having at least one bladder or voiding symptom (e.g., frequency, urgency, dysuria) with bacteriuria (≥105 colony forming units/mL of no more than 2 urinary tract pathogens). 4. Stone in the renal pelvis, ureter, bladder or urethra 5. Pregnant females 6. Solitary kidney, transplant kidney, urinary diversion, medullary sponge kidney 7. Anatomic abnormality of the kidney (e.g., horseshoe kidney) 8. Parents/guardians or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Healthcare-related life disruption | Every 3 months (+/- 14 days) from date of consent (observational cohort) or randomization (randomized cohort) until 24 months. | Composite of: Stone-specific emergency department (ED)/urgent care visits, stone-specific hospitalizations and surgery for stones for new symptoms (Shockwave Lithotripsy (SWL) and Ureteroscopy (URS)) as reported via Patient Centered Outcomes Research Network (PCORnet) Common Data Model (CDM) query and participant self-report on REDCap. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Patient reported physical and emotional health (Pain Interference) | Every 3 months (+/- 14 days) from date of consent (observational cohort) or randomization (randomized cohort) until 24 months. | Patient-Reported Outcomes Measurement Information System (PROMIS) Pain Interference (PI) is a standardized, self-reported assessment tool developed by the National Institutes of Health (NIH). It measures how much pain hinders a person's physical, mental, social, and recreational activities, as well as their sleep and enjoyment of life. It uses a standardized T-score (average is 50; standard deviation is 10) to reflect the level of disruption. Higher numbers mean more pain interference (pain is having a greater negative impact on your daily life, making it harder to participate in work, social, and physical activities). The study team is using the validated versions for adults (18 years and older at time of consent), children (ages 8-17 years at time of consent), and parent-proxy versions for caregivers of children ages (6-7 years at time of consent). This information will be captured via patient report in REDCap. |
| Patient reported physical and emotional health (Anxiety) | Every 3 months (+/- 14 days) from date of consent (observational cohort) or randomization (randomized cohort) until 24 months. | Patient-Reported Outcomes Measurement Information System (PROMIS) Anxiety is a standardized, self-reported assessment tool developed by the National Institutes of Health (NIH). It measures the severity of fear, worry, dread, hyperarousal, and physical stress symptoms over the past seven days on a 5-point scale (ranging from never to always). Raw scores are converted into a standardized T-score with an average of 50 and a standard deviation of 10. A higher T-score indicates a higher severity of anxiety. The study team is using validated versions for adults (18 years and older at time of consent), children (ages 8-17 years at time of consent), and parent-proxy versions for caregivers of children ages (6-7 years at time of consent). This information will be captured via patient report in REDCap. |
| Patient reported physical and emotional health (Stone Quality of Life) | Every 3 months (+/- 14 days) from date of consent (observational cohort) or randomization (randomized cohort) until 24 months. | The Wisconsin Stone Quality of Life questionnaire (WISQOL) is a disease-specific health-related quality of life (HRQOL) instrument designed to assess the impact on patients of stones in the urinary tract. Each item is scored on a 5-point Likert scale, yielding a total raw score of 28 to 140. Higher scores reflect a better health-related quality of life. Raw scores are typically converted to a standardized 0-100 scale. This information will be captured via patient report on REDCap. |
| Stone passage | Every 3 months (+/- 14 days) from date of consent (observational cohort) or randomization (randomized cohort) until 24 months. Reporting total after 2 years. | Patient Reported Survey: Number of self-reported stone passages (A kidney stone episode is passing a stone that caused pain or other bothersome symptoms or having surgery to remove a stone.) This information will be captured via patient report on REDCap. |
| Lost productivity (Missed School) | Every 3 months (+/- 14 days) from date of consent (observational cohort) or randomization (randomized cohort) until 24 months. | Patient Reported Survey: Number of days the patient (for patients under 18 years old at time of consent) missed school due to kidney stones or kidney stone-related care (0 days, 1-2 days, 3-5 days, 6-10 days, more than 10 days) in the last 3 months. This information will be captured via patient report in REDCap. |
| Lost productivity (Caregiver Missed Work) | Every 3 months (+/- 14 days) from date of consent (observational cohort) or randomization (randomized cohort) until 24 months. | Patient Reported Survey: Number of days the patient's caregiver(s) (for patients \<18 years old at time of consent) missed work due to kidney stones or kidney stone-related care (0 days, 1-2 days, 3-5 days, 6-10 days, more than 10 days) in the last 3 months. This information will be captured via patient report on REDCap. |
| Lost productivity (Patient Missed Work) | Every 3 months (+/- 14 days) from date of consent (observational cohort) or randomization (randomized cohort) until 24 months. | Patient Reported Survey: Number of days the patient missed work (for patients 18 years and older at time of consent) due to kidney stones or kidney stone-related care (0 days, 1-2 days, 3-5 days, 6-10 days, More than 10 days) in the last 3 months. This information will be captured via patient report in REDCap. |
Countries
United States
Contacts
CONTACTRebecca D McCune, BS, MS
CONTACTBrian J Augelli, BS
PRINCIPAL_INVESTIGATORGregory E Tasian, MD, MSc, MSCE
Children's Hospital of Philadelphia
Outcome results
None listed