Clinical trial NCT04884945: Pediatric Robotic Versus Open Pyeloplasty
Official title: Pediatric Robotic Versus Open Pyeloplasty: A Pilot Randomized Control Study
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: NA
Conditions: Pediatric Urology
Ureteropelvic Junction Obstruction
Keywords: patient centered outcomes
Lead sponsor: Indiana University
Sponsor class: OTHER
Collaborators: name: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
First posted: 2021-05-13
Enrollment: 11
Sex: ALL
Minimum age (years): 2
Maximum age (years): 8
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: For Aim 2: For the RCT, patients are randomized to either RALP or OP by using a pool of 20 cards of which 10 say "robotic surgery" and 10 say "open surgery.
Primary purpose: OTHER

Brief summary: The objective is conduct a pilot randomized controlled trial comparing robotic and open techniques for surgical correction of congenital UPJ obstruction (pyeloplasty) in pediatric patients. This study will serve as a proof of concept trial to demonstrate feasibility of recruiting pediatric patients to participate in a randomized study for surgical procedures and delineate patient-centered outcomes. Should this study prove randomization is feasible, a randomized comparative effectiveness trial with sufficient power to determine whether open or robotic-assisted pyeloplasty has superior patient-centered outcomes will be pursued.

Detailed description: Technological innovations are an ongoing reality in clinical medicine, most dramatically in the operating room. Each advance is often heralded as an improvement in patient care, promising to correct deficiencies and advance standard operative procedures. Unfortunately, these new technologies often lack sufficient evidence to support such claims. Robotic assisted laparoscopic (RAL) surgery continues to gain popularity among pediatric surgical specialists, particularly for reconstructive procedures such as correction of ureteropelvic junction (UPJ) obstruction or vesicoureteral reflux (VUR).In these operations, where traditional open surgery yields a nearly 95% clinical success rate it is difficult to demonstrate improvement with a new surgical technique. Despite these impressive outcomes with the open approach many pediatric urologists are shifting towards newer RAL approaches, claiming faster recovery and improved cosmesis with a nominal increase in surgical cost.

There is a striking dearth of comparative effectiveness research in pediatric surgical specialties, especially given the wide range of available therapies for a variety of surgical problems. Conducting randomized controlled trials of surgical interventions can be very difficult, especially in the pediatric population.

Currently, all studies of pediatric robotic surgery have focused on traditional outcomes, e.g., length of stay, pain scores, pain medication usage, scar perception, cost analysis, and capital gains benefits. While these measures are important to study, the investigators believe that researchers must also focus on patients' treatment experiences or patients' evaluations of their own outcomes. By focusing on these 'patient-centered outcomes' the investigators will be able to improve outcomes from surgical procedures in ways that are most important to patients and their families.

We propose a pilot randomized controlled trial comparing robotic and open techniques for surgical correction of congenital UPJ obstruction (pyeloplasty) in pediatric patients. This study will serve as a proof of concept trial to demonstrate feasibility of recruiting pediatric patients to participate in a randomized study for surgical procedures and delineate patient-centered outcomes. Should this study prove randomization is feasible, the investigators will seek funding to conduct a large, randomized comparative effectiveness trial with sufficient power to determine whether open or robotic-assisted pyeloplasty has superior patient-centered outcomes.

The investigators propose to accomplish these goals with the following Specific Aims:

Aim 1: Utilize novel patient-centered research methods to:

* Identify outcomes of importance to patients and families related to pediatric pyeloplasty.
* Determine patient and family preferences regarding acceptability of randomization and blinding of pediatric surgical patients and construct a recruitment approach to achieve enrollment.

Aim 2: Conduct a randomized pilot study with goal of enrolling ten to twenty pediatric patients (age 2 - 8 years) to either open or robotic pyeloplasty for treatment of primary UPJ obstruction.

Sub Aim 2a: Collect preliminary data regarding the patient-centered outcomes identified in Aim 1.

Sub Aim 2b: Evaluate the effectiveness of the recruitment approach for randomized surgical studies developed in Aim 1.

The investigators believe that the use of innovative and novel patient-centered research methods will lead to a significant breakthrough in developing a recruitment approach that is both acceptable to pediatric patients/families and optimizes participant willingness to participate in RCTs of surgical interventions. Additionally these same innovative research methods will allows the investigators to explore what outcomes are meaningful to patients and families, which will hopefully allow the care team to counsel patients and families in a way that maximizes their chances of achieving patient- and family-centered goals.

Eligibility criteria: For Aim 2:

Inclusion Criteria:

* Primary UPJ obstruction diagnosed by scan and presenting to the pediatric urology clinic for evaluation
* Patient between age 2 - 8 years old

Exclusion Criteria:

* Serious comorbidities (cardiovascular or respiratory disease, or other congenital anomalies requiring surgical intervention).
* BMI greater than the 95th %tile for age.

Primary outcomes:
measure: Length of Post Op Inpatient Hospital Stay Measured in Days
timeFrame: surgery to discharge
measure: Number of Doses of Narcotic Pain Medication In the First 24 Hours Post Op
timeFrame: Surgery to the first 24 hours post op
measure: Total Dose of Narcotic Pain Medication In the First 24 Hours Post Op in Morphine Milligram Equivalents (MME)
timeFrame: Surgery to the first 24 hours post op
measure: Grade of Surgical Complications up to 1 Year Post op Using Clavian-Dindo Classification
timeFrame: up to 1 year post up
measure: Pain Score Measured Using The FLACC at 2 Week Post Op Follow Up
timeFrame: 1 week post op follow up
measure: Length Of Time For Patient To Return To Normal Routine After Surgery Measured in Days at 2 Week Post Follow Up
timeFrame: 2 week post op follow up
measure: Length Of Time Parent Was Out Of Work After Child's Surgery Measured in Days at 2 Week Post Follow Up
timeFrame: 2 week post op follow up
measure: Satisfaction With Scar Appearance Measured Using the Patient Scar Assessment Questionnaire at 3 Month Follow Up Visit
timeFrame: up to 3 month follow up visit (18 weeks post op)
measure: Satisfaction With Symptoms Measured Using the Patient Scar Assessment Questionnaire at 3 Month Follow Up Visit
timeFrame: up to 3 month follow up visit (18 weeks post op)
measure: Quality of Life Measured Using The Glasgow Children's Benefit Inventory at 3 Month Follow Up Visit
timeFrame: 3 month follow up visit
Secondary outcomes:
Other outcomes:
Locations:
city: Indianapolis
country: United States
facility: Riley Children's Hospital
state: Indiana
Study officials:
affiliation: Riley Children's Health, Indiana University Health, Pediatric Urology
name: Benjamin Whittam, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Monn MF, Bahler CD, Schneider EB, Whittam BM, Misseri R, Rink RC, Sundaram CP. Trends in robot-assisted laparoscopic pyeloplasty in pediatric patients. Urology. 2013 Jun;81(6):1336-41. doi: 10.1016/j.urology.2013.01.025. Epub 2013 Mar 19.
citation: Liu DB, Ellimoottil C, Flum AS, Casey JT, Gong EM. Contemporary national comparison of open, laparoscopic, and robotic-assisted laparoscopic pediatric pyeloplasty. J Pediatr Urol. 2014 Aug;10(4):610-5. doi: 10.1016/j.jpurol.2014.06.010. Epub 2014 Jul 11.
citation: Steffens J, Langen PH, Haben B, Hiebl R, Steffens L, Polsky MS. Politano-leadbetter ureteroneocystostomy. A 30-year experience. Urol Int. 2000;65(1):9-14. doi: 10.1159/000064827.
citation: Shaul DB, Cunningham JA, Lowe P, Skaist LB, Hardy BE. Infant pyeloplasty is a low-risk procedure. J Pediatr Surg. 1994 Feb;29(2):343-7. doi: 10.1016/0022-3468(94)90345-x.
citation: Hendren WH, Radhakrishnan J, Middleton AW Jr. Pediatric pyleoplasty. J Pediatr Surg. 1980 Apr;15(2):133-44. doi: 10.1016/s0022-3468(80)80004-2.
citation: Salem YH, Majd M, Rushton HG, Belman AB. Outcome analysis of pediatric pyeloplasty as a function of patient age, presentation and differential renal function. J Urol. 1995 Nov;154(5):1889-93.
citation: Behan JW, Kim SS, Dorey F, De Filippo RE, Chang AY, Hardy BE, Koh CJ. Human capital gains associated with robotic assisted laparoscopic pyeloplasty in children compared to open pyeloplasty. J Urol. 2011 Oct;186(4 Suppl):1663-7. doi: 10.1016/j.juro.2011.04.019.
citation: Lee RS, Retik AB, Borer JG, Peters CA. Pediatric robot assisted laparoscopic dismembered pyeloplasty: comparison with a cohort of open surgery. J Urol. 2006 Feb;175(2):683-7; discussion 687. doi: 10.1016/S0022-5347(05)00183-7.
citation: Cundy TP, Harling L, Hughes-Hallett A, Mayer EK, Najmaldin AS, Athanasiou T, Yang GZ, Darzi A. Meta-analysis of robot-assisted vs conventional laparoscopic and open pyeloplasty in children. BJU Int. 2014 Oct;114(4):582-94. doi: 10.1111/bju.12683.
citation: Barbosa JA, Barayan G, Gridley CM, Sanchez DC, Passerotti CC, Houck CS, Nguyen HT. Parent and patient perceptions of robotic vs open urological surgery scars in children. J Urol. 2013 Jul;190(1):244-50. doi: 10.1016/j.juro.2012.12.060. Epub 2012 Dec 28.
citation: Bennett WE Jr, Whittam BM, Szymanski KM, Rink RC, Cain MP, Carroll AE. Validated cost comparison of open vs. robotic pyeloplasty in American children's hospitals. J Robot Surg. 2017 Jun;11(2):201-206. doi: 10.1007/s11701-016-0645-1. Epub 2016 Oct 20.
citation: Ferreira LM. Surgical randomized controlled trials: reflection of the difficulties. Acta Cirurgica Brasileira 2005;19(Suppl 1):2-3.
citation: McCulloch P, Taylor I, Sasako M, Lovett B, Griffin D. Randomised trials in surgery: problems and possible solutions. BMJ. 2002 Jun 15;324(7351):1448-51. doi: 10.1136/bmj.324.7351.1448. No abstract available.
citation: Caldwell PH, Murphy SB, Butow PN, Craig JC. Clinical trials in children. Lancet. 2004 Aug 28-Sep 3;364(9436):803-11. doi: 10.1016/S0140-6736(04)16942-0.
citation: Curry JI, Reeves B, Stringer MD. Randomized controlled trials in pediatric surgery: could we do better? J Pediatr Surg. 2003 Apr;38(4):556-9. doi: 10.1053/jpsu.2003.50121.
citation: Rosenthal R, Kasenda B, Dell-Kuster S, von Elm E, You J, Blumle A, Tomonaga Y, Saccilotto R, Amstutz A, Bengough T, Meerpohl JJ, Stegert M, Tikkinen KA, Neumann I, Carrasco-Labra A, Faulhaber M, Mulla S, Mertz D, Akl EA, Bassler D, Busse JW, Ferreira-Gonzalez I, Lamontagne F, Nordmann A, Gloy V, Olu KK, Raatz H, Moja L, Ebrahim S, Schandelmaier S, Sun X, Vandvik PO, Johnston BC, Walter MA, Burnand B, Schwenkglenks M, Hemkens LG, Bucher HC, Guyatt GH, Briel M. Completion and Publication Rates of Randomized Controlled Trials in Surgery: An Empirical Study. Ann Surg. 2015 Jul;262(1):68-73. doi: 10.1097/SLA.0000000000000810.
citation: Casella DP, Fox JA, Schneck FX, Cannon GM, Ost MC. Cost analysis of pediatric robot-assisted and laparoscopic pyeloplasty. J Urol. 2013 Mar;189(3):1083-6. doi: 10.1016/j.juro.2012.08.259. Epub 2012 Sep 24.
citation: Yu HY, Hevelone ND, Lipsitz SR, Kowalczyk KJ, Hu JC. Use, costs and comparative effectiveness of robotic assisted, laparoscopic and open urological surgery. J Urol. 2012 Apr;187(4):1392-8. doi: 10.1016/j.juro.2011.11.089. Epub 2012 Feb 16.
citation: Cundy TP, Shetty K, Clark J, Chang TP, Sriskandarajah K, Gattas NE, Najmaldin A, Yang GZ, Darzi A. The first decade of robotic surgery in children. J Pediatr Surg. 2013 Apr;48(4):858-65. doi: 10.1016/j.jpedsurg.2013.01.031.
citation: Tanaka ST, Grantham JA, Thomas JC, Adams MC, Brock JW 3rd, Pope JC 4th. A comparison of open vs laparoscopic pediatric pyeloplasty using the pediatric health information system database--do benefits of laparoscopic approach recede at younger ages? J Urol. 2008 Oct;180(4):1479-85. doi: 10.1016/j.juro.2008.06.044. Epub 2008 Aug 16.
citation: Fowler FJ Jr, Barry MJ, Lu-Yao G, Wasson J, Roman A, Wennberg J. Effect of radical prostatectomy for prostate cancer on patient quality of life: results from a Medicare survey. Urology. 1995 Jun;45(6):1007-13; discussion 1013-5. doi: 10.1016/s0090-4295(99)80122-8.
citation: Resnick MJ, Barocas DA, Morgans AK, Phillips SE, Koyama T, Albertsen PC, Cooperberg MR, Goodman M, Greenfield S, Hamilton AS, Hoffman KE, Hoffman RM, Kaplan SH, McCollum D, Paddock LE, Stanford JL, Stroup AM, Wu XC, Penson DF. The Evolution of Self-Reported Urinary and Sexual Dysfunction over the Last Two Decades: Implications for Comparative Effectiveness Research. Eur Urol. 2015 Jun;67(6):1019-1025. doi: 10.1016/j.eururo.2014.08.035. Epub 2014 Aug 28.
citation: Morin L, Cendron M, Crombleholme TM, Garmel SH, Klauber GT, D'Alton ME. Minimal hydronephrosis in the fetus: clinical significance and implications for management. J Urol. 1996 Jun;155(6):2047-9. doi: 10.1016/s0022-5347(01)66102-0.
citation: Persky L, Krause JR, Boltuch RL. Initial complications and late results in dismembered pyeloplasty. J Urol. 1977 Jul;118(1 Pt 2):162-5. doi: 10.1016/s0022-5347(17)57936-7.
citation: Psooy K, Pike JG, Leonard MP. Long-term followup of pediatric dismembered pyeloplasty: how long is long enough? J Urol. 2003 May;169(5):1809-12; discussion 1812; author reply 1812. doi: 10.1097/01.ju.0000055040.19568.ea.
citation: Riachy E, Cost NG, Defoor WR, Reddy PP, Minevich EA, Noh PH. Pediatric standard and robot-assisted laparoscopic pyeloplasty: a comparative single institution study. J Urol. 2013 Jan;189(1):283-7. doi: 10.1016/j.juro.2012.09.008. Epub 2012 Nov 20.
citation: Penn HA, Gatti JM, Hoestje SM, DeMarco RT, Snyder CL, Murphy JP. Laparoscopic versus open pyeloplasty in children: preliminary report of a prospective randomized trial. J Urol. 2010 Aug;184(2):690-5. doi: 10.1016/j.juro.2010.03.062. Epub 2010 Jun 19.
citation: Mei H, Pu J, Yang C, Zhang H, Zheng L, Tong Q. Laparoscopic versus open pyeloplasty for ureteropelvic junction obstruction in children: a systematic review and meta-analysis. J Endourol. 2011 May;25(5):727-36. doi: 10.1089/end.2010.0544. Epub 2011 Apr 8.
citation: Piaggio LA, Franc-Guimond J, Noh PH, Wehry M, Figueroa TE, Barthold J, Gonzalez R. Transperitoneal laparoscopic pyeloplasty for primary repair of ureteropelvic junction obstruction in infants and children: comparison with open surgery. J Urol. 2007 Oct;178(4 Pt 2):1579-83. doi: 10.1016/j.juro.2007.03.159. Epub 2007 Aug 16.
citation: Yohannes P, Rotariu P, Pinto P, Smith AD, Lee BR. Comparison of robotic versus laparoscopic skills: is there a difference in the learning curve? Urology. 2002 Jul;60(1):39-45; discussion 45. doi: 10.1016/s0090-4295(02)01717-x.
citation: Lim PC, Kang E, Park DH. A comparative detail analysis of the learning curve and surgical outcome for robotic hysterectomy with lymphadenectomy versus laparoscopic hysterectomy with lymphadenectomy in treatment of endometrial cancer: a case-matched controlled study of the first one hundred twenty two patients. Gynecol Oncol. 2011 Mar;120(3):413-8. doi: 10.1016/j.ygyno.2010.11.034. Epub 2010 Dec 30.
citation: Sorensen MD, Delostrinos C, Johnson MH, Grady RW, Lendvay TS. Comparison of the learning curve and outcomes of robotic assisted pediatric pyeloplasty. J Urol. 2011 Jun;185(6 Suppl):2517-22. doi: 10.1016/j.juro.2011.01.021. Epub 2011 Apr 28.
citation: Chandra V, Nehra D, Parent R, Woo R, Reyes R, Hernandez-Boussard T, Dutta S. A comparison of laparoscopic and robotic assisted suturing performance by experts and novices. Surgery. 2010 Jun;147(6):830-9. doi: 10.1016/j.surg.2009.11.002. Epub 2009 Dec 31.
citation: Varda BK, Johnson EK, Clark C, Chung BI, Nelson CP, Chang SL. National trends of perioperative outcomes and costs for open, laparoscopic and robotic pediatric pyeloplasty. J Urol. 2014 Apr;191(4):1090-5. doi: 10.1016/j.juro.2013.10.077. Epub 2013 Oct 25.
citation: Dangle PP, Kearns J, Anderson B, Gundeti MS. Outcomes of infants undergoing robot-assisted laparoscopic pyeloplasty compared to open repair. J Urol. 2013 Dec;190(6):2221-6. doi: 10.1016/j.juro.2013.07.063. Epub 2013 Aug 1.
citation: Srougi V, Yorioka M, Sanchez DC, Onal B, Houck CS, Nguyen HT. The feasibility of robotic urologic surgery in infants and toddlers. J Pediatr Urol. 2013 Dec;9(6 Pt B):1198-203. doi: 10.1016/j.jpurol.2013.05.011. Epub 2013 Jun 17.
citation: Yee DS, Shanberg AM, Duel BP, Rodriguez E, Eichel L, Rajpoot D. Initial comparison of robotic-assisted laparoscopic versus open pyeloplasty in children. Urology. 2006 Mar;67(3):599-602. doi: 10.1016/j.urology.2005.09.021. Epub 2006 Feb 28.
citation: Greenfield S, Kaplan SH. Building useful evidence: changing the clinical research paradigm to account for comparative effectiveness research. J Comp Eff Res. 2012 May;1(3):263-70. doi: 10.2217/CER.12.23.
citation: Sequist TD, Schneider EC, Anastario M, Odigie EG, Marshall R, Rogers WH, Safran DG. Quality monitoring of physicians: linking patients' experiences of care to clinical quality and outcomes. J Gen Intern Med. 2008 Nov;23(11):1784-90. doi: 10.1007/s11606-008-0760-4. Epub 2008 Aug 28.
citation: Freilich DA, Penna FJ, Nelson CP, Retik AB, Nguyen HT. Parental satisfaction after open versus robot assisted laparoscopic pyeloplasty: results from modified Glasgow Children's Benefit Inventory Survey. J Urol. 2010 Feb;183(2):704-8. doi: 10.1016/j.juro.2009.10.040. Epub 2009 Dec 21.
citation: Ostlie DJ, St Peter SD. The current state of evidence-based pediatric surgery. J Pediatr Surg. 2010 Oct;45(10):1940-6. doi: 10.1016/j.jpedsurg.2010.05.008.
citation: Chapman SJ, Shelton B, Mahmood H, Fitzgerald JE, Harrison EM, Bhangu A. Discontinuation and non-publication of surgical randomised controlled trials: observational study. BMJ. 2014 Dec 9;349:g6870. doi: 10.1136/bmj.g6870.
citation: Sanematsu H, Wiehe SE. Insight 2: Engaging the health humanities. 2013
citation: Sanematsu H, Wiehe S. Learning to look: Design in health services research. Touchpoint 2014
citation: Abraham NS, Hewett P, Young JM, Solomon MJ. Non-entry of eligible patients into the Australasian Laparoscopic Colon Cancer Study. ANZ J Surg. 2006 Sep;76(9):825-9. doi: 10.1111/j.1445-2197.2006.03878.x.
citation: Jorgensen LN, Rosenberg J, Al-Tayar H, Assaadzadeh S, Helgstrand F, Bisgaard T. Randomized clinical trial of single- versus multi-incision laparoscopic cholecystectomy. Br J Surg. 2014 Mar;101(4):347-55. doi: 10.1002/bjs.9393.
citation: Sanematsu H, Hong Y, Cole L. Listening through Seeing: Using Design Methods to Learn about the Health Perceptions of Garden on the Go Customors. Indianapolis: 2014
citation: Bennet A, Eglash R, Krishnamoorthy M, Rarieya M. Audience as Co-Designer: Participatory Design of HIV/AIDS Awareness & Prevention Posters in Kenya. In: Design Studies. New York: 2006. p. 179-97
citation: Sanematsu H. 53. Fun with Facebook: The Impact of Focus Groups on the Development of Awareness Campaigns for Adolescent Health. Journal of Adolescent Health 2011;48(2):S44-5
citation: Bate P, Robert G. Experience-based design: from redesigning the system around the patient to co-designing services with the patient. Qual Saf Health Care. 2006 Oct;15(5):307-10. doi: 10.1136/qshc.2005.016527.
citation: Clavien PA, Barkun J, de Oliveira ML, Vauthey JN, Dindo D, Schulick RD, de Santibanes E, Pekolj J, Slankamenac K, Bassi C, Graf R, Vonlanthen R, Padbury R, Cameron JL, Makuuchi M. The Clavien-Dindo classification of surgical complications: five-year experience. Ann Surg. 2009 Aug;250(2):187-96. doi: 10.1097/SLA.0b013e3181b13ca2.
citation: Durani P, McGrouther DA, Ferguson MW. The Patient Scar Assessment Questionnaire: a reliable and valid patient-reported outcomes measure for linear scars. Plast Reconstr Surg. 2009 May;123(5):1481-1489. doi: 10.1097/PRS.0b013e3181a205de.