Top

International Journal of Colorectal Disease

Published in:

Open Access 01-04-2018 | Clinical Study Protocol

Sacral neuromodulation versus personalized conservative treatment in patients with idiopathic slow-transit constipation: study protocol of the No.2-trial, a multicenter open-label randomized controlled trial and cost-effectiveness analysis

Authors: S. C. M. Heemskerk, A. H. Rotteveel, M. A. Benninga, C. I. M. Baeten, A. A. M. Masclee, J. Melenhorst, S. M. J. van Kuijk, C. D. Dirksen, S. O. Breukink

Published in: International Journal of Colorectal Disease | Issue 4/2018

Abstract

Purpose

The evidence regarding the (cost-)effectiveness of sacral neuromodulation (SNM) in patients with therapy-resistant idiopathic slow-transit constipation is of suboptimal quality. The Dutch Ministry of Health, Welfare and Sports has granted conditional reimbursement for SNM treatment. The objective is to assess the effectiveness, cost-effectiveness, and budget impact of SNM compared to personalized conservative treatment (PCT) in patients with idiopathic slow-transit constipation refractory to conservative treatment.

Methods

This study is an open-label, multicenter randomized controlled trial. Patients aged 14 to 80 with slow-transit constipation, a defecation frequency (DF) < 3 per week and meeting at least one other Rome-IV criterion, are eligible. Patients with obstructed outlet, irritable bowel syndrome, bowel pathology, or rectal prolapse are excluded. Patients are randomized to SNM or PCT. The primary outcome is success at 6 months (DF ≥ 3 a week), requiring a sample size of 64 (α = 0.05, β = 0.80, 30% difference in success). Secondary outcomes are straining, sense of incomplete evacuation, constipation severity, fatigue, constipation specific and generic quality of life, and costs at 6 months. Long-term costs and effectiveness will be estimated by a decision analytic model. The time frame is 57 months, starting October 2016. SNM treatment costs are funded by the Dutch conditional reimbursement program, research costs by Medtronic.

Conclusions

The results of this trial will be used to make a final decision regarding reimbursement of SNM from the Dutch Health Care Package in this patient group.

Trial registration

This trial is registered at clinicaltrials.gov, identifier NCT02961582, on 12 October 2016.

Available only for authorised users

Lacy BE, Mearin F, Chang L, Chey WD, Lembo AJ, Simren M, Spiller R (2016) Bowel disorders. Gastroenterology 150(6):1393–1407. https://doi.org/10.1053/j.gastro.2016.02.031 CrossRef

Hyams JS, Di Lorenzo C, Saps M, Shulman RJ, Staiano A, van Tilburg M (2016) Childhood functional gastrointestinal disorders: child/adolescent. Gastroenterology 150(6):1456–1468. https://doi.org/10.1053/j.gastro.2016.02.015 CrossRef

Suares NC, Ford AC (2011) Prevalence of, and risk factors for, chronic idiopathic constipation in the community: systematic review and meta-analysis. Am J Gastroenterol 106(9):1582–1591. https://doi.org/10.1038/ajg.2011.164 CrossRefPubMed

Mugie SM, Benninga MA, Di Lorenzo C (2011) Epidemiology of constipation in children and adults. Best Pract Res Clin Gastroenterol 25:3–18. https://doi.org/10.1016/j.bpg.2010.12.010 CrossRefPubMed

Dik VK, Siersema PD, Joseph A, Hodgkins P, Smeets HM, van Oijen MGH (2014) Constipation-related direct medical costs in 16 887 patients newly diagnosed with chronic constipation. Eur J Gastroenterol Hepatol 26:1260–1266. https://doi.org/10.1097/MEG.0000000000000167 CrossRefPubMedPubMedCentral

Knowles CH, Martin JE (2000) Slow transit constipation: a model of human gut dysmotility. Review of possible aetiologies. Neurogastroenterol Motil 12(2):181–196CrossRefPubMed

Frattini JC, Nogueras JJ (2008) Slow transit constipation: a review of a colonic functional disorder. Clin Colon Rectal Surg 21(2):146–152. https://doi.org/10.1055/s-2008-1075864 CrossRefPubMedPubMedCentral

Mugie SM, Di Lorenzo C, Benninga MA (2011) Constipation in childhood. Nat Rev Gastroenterol Hepatol 8(9):502–511. https://doi.org/10.1038/nrgastro.2011.130 CrossRefPubMed

Bove A, Bellini M, Battaglia E, Bocchini R, Gambaccini D, Bove V, Pucciani F, Altomare DF, Dodi G, Sciaudone G, Falletto E, Piloni V (2012) Consensus statement AIGO/SICCR diagnosis and treatment of chronic constipation and obstructed defecation (Part II:Treatment). World J Gastroenterol 18(36):4994–5013. https://doi.org/10.3748/wjg.v18.i36.4994 CrossRefPubMedPubMedCentral

10.

Tabbers MM, Di Lorenzo C, Berger MY, Faure C, Langendam MW, Nurko S, Staiano A, Vandenplas Y, Benninga M (2014) Evaluation and treatment of functional constipation in infants and children: evidence-based recommendations from ESPGHAN and NASPGHAN. J Pediatr Gastroenterol Nutr 58(2):258–274. https://doi.org/10.1097/MPG.0000000000000266 CrossRefPubMed

11.

Kamm MA (2006) Clinical case: chronic constipation. Gastroenterology 131:233–239. https://doi.org/10.1053/j.gastro.2006.05.027 CrossRefPubMed

12.

Kuizenga-Wessel S (2017) Functional defecation disorders in children: associated comorbidity and advances in management. Dissertation. Universiteit van Amsterdam, Amsterdam

13.

Basilico G, Coletta M (2013) Chronic constipation: a critical review. Dig Liver Dis 45:886–893. https://doi.org/10.1016/j.dld.2013.03.016 CrossRef

14.

Belsey J, Greenfield S, Candy D, Geraint M (2010) Systematic review: impact of constipation on quality of life in adults and children. Aliment Pharmacol Ther 31(9):938–949. https://doi.org/10.1111/j.1365-2036.2010.04273.x PubMed

15.

Sharma A, Bussen D, Herold A, Jayne D (2013) Review of sacral neuromodulation for management of constipation. Surg Innov 20(6):614–624. https://doi.org/10.1177/1553350613475882 CrossRefPubMed

16.

Brazzelli M, Murray A, Fraser C (2006) Efficacy and safety of sacral nerve stimulation for urinary urge incontinence: a systematic review. J Urol 175:835–841. https://doi.org/10.1016/S0022-5347(05)00326-5 CrossRefPubMed

17.

Jarett MED, Mowatt G, Galzener CMA, Fraser C, Nichols RJ, Grant AM, Kamm MA (2004) Systematic review of sacral nerve stimulation for faecal incontinence and constipation. Br J Surg 91:1559–1569. https://doi.org/10.1002/bjs.4796 CrossRef

18.

Norderval S, Rydningen M, Lindsetmo RO, Lein D, Vonen B (2011) Sacral nerve stimulation. Tidsskrift for Den norske legeforening 12(131):1190–1193CrossRef

19.

Patton V, Stewart P, Lubowski DZ, Cook IJ, Dinning PG (2016) Sacral nerve stimulation fails to offer long-term benefit in patients with slow-transit constipation. Dis Colon rectum 59(9):878–885. https://doi.org/10.1097/dcr.0000000000000653

20.

Zerbib F, Siproudhis L, Lehur PA, Germain C, Mion F, Leroi AM, Coffin B, Le Sidaner A, Vitton V, Bouyssou-Cellier C, Chene G (2016) Randomized clinical trial of sacral nerve stimulation for refractory constipation. Br J Surg 104:205–213. https://doi.org/10.1002/bjs.10326 CrossRefPubMed

21.

Kamm MA, Dudding TC, Melenhorst J, Jarrett M, Wang Z, Buntzen JC, Laurberg S, Rosen H, Vaizey CJ, Matzel K, Baeten C (2010) Sacral nerve stimulation for intractable constipation. Gut 59:333–340CrossRefPubMed

22.

Ortiz H, de Miguel M, Rinaldi M, Oteiza F, Altomare DF (2012) Functional outcome of sacral nerve stimulation in patients with severe constipation. Dis Colon rectum 55:876–880. https://doi.org/10.1097/DCR.0b013e31825bc9af

23.

Govaert B, Maeda Y, Alberga J, Buntzen S, Laurberg S, Baeten CG (2012) Medium-term outcome of sacral nerve modulation for constipation. Dis Colon rectum 55(1):26–31. https://doi.org/10.1097/DCR.0b013e31823898a5

24.

Thomas GP, Dudding TC, Rahbour G, Nicholls RJ, Vaizey CJ (2013) Sacral nerve stimulation for constipation. Br J Surg 100:174–181. https://doi.org/10.1002/bjs.8944 CrossRefPubMed

25.

Thaha MA, Abukar AA, Thin NN, Ramsanahie A, Knowles CH (2015) Sacral nerve stimulation for faecal incontinence and constipation in adults. Cochrane Database Syst Rev 8. https://doi.org/10.1002/14651858.CD004464.pub3

26.

Zorginstituut Nederland (2014) Standpunt sacrale neurostimulatie bij kinderen en volwassen met therapieresistente functionele obstipatie (Report on sacral neurostimulation in children and adults with therapy-resistant functional constipation). Zorginstituut Nederland, Diemen

27.

Rijksoverheid (2016) Zenuwstimulatie bij ernstige chronische verstopping van de darmen voorwaardelijk toegelaten tot het basispakket. https://www.rijksoverheid.nl/actueel/nieuws/2016/09/03/zenuwstimulatie-bij-ernstige-chronische-verstopping-van-de-darmen-voorwaardelijk-toegelaten-tot-het-basispakket. Accessed February 2017

28.

Bouchoucha M, Devroede G, Bon C, Raynaud JJ, Bejou B, Benamouzig R (2015) How many segments are necessary to characterize delayed colonic transit time? Int J Color Dis 30(10):1381–1389. https://doi.org/10.1007/s00384-015-2277-8 CrossRef

29.

Martellucci J (2015) The technique of sacral nerve modulation. Color Dis 17(4):O88–O94. https://doi.org/10.1111/codi.12900 CrossRef

30.

Agachan F, Chen T, Pfeifer J, Reissman P, Wexner SD (1996) A constipation scoring system to simplify evaluation and management of constipated patients. Dis Colon rectum 39(6):681–685

31.

Alberts M, Smets EMA, Vercoulen JHMM, Garssen B, Bleijenberg G (1997) ‘Verkorte vermoeidheidsvragenlijst’: een praktisch hulpmiddel bij het scoren van vermoeidheid. Nederlands Tijdschrift voor de Geneeskunde 141(31):1526–1530

32.

Marquis P, De La Loge C, Dubois D, McDermott A, Chassany O (2005) Development and validation of the Patient Assessment of Constipation Quality of Life questionnaire. Scand J Gastroenterol 40(5):540–551. https://doi.org/10.1080/00365520510012208 CrossRefPubMed

33.

Herdman M, Gudex C, Lloyd A, Janssen M, Kind P, Parkin D, Bonsel G, Badia X (2011) Development and preliminary testing of the new five-level version of EQ-5D (EQ-5D-5L). Qual Life Res 20(10):1727–1736. https://doi.org/10.1007/s11136-011-9903-x CrossRefPubMedPubMedCentral

34.

Al-Janabi H, Flynn TN, Coast J (2012) Development of a self-report measure of capability wellbeing for adults: the ICECAP-A. Qual Life Res 21:167–176. https://doi.org/10.1007/s11136-011-9927-2 CrossRefPubMed

35.

van Reenen M, Janssen B, Oppe M, Kreimeier S, Greiner W (2014) EQ-5D-Y User Guide. EuroQoL Group Rotterdam

36.

Ravens-Sieberer U, Auquier P, Erhart M, Gosch A, Rajmil L, Bruil J (2007) The Kidscreen-27 quality of life measure for children and adolescent: psychometric results from a cross-cultural survey in 13 European countries. Qual Life Res 16:1347–1356. https://doi.org/10.1007/s11136-007-9240-2 CrossRefPubMed

37.

Robitail S, Ravens-Sieberer U, Simeoni M-C, Rajmil L, Bruil J, Power M, Duer W, Cloetta B, Czemy L, Mazur J, Czimbalmos A, Tountas Y, Hagquist C, Kilroe J, Auquier P (2007) Testing the structural and cross-cultural validity of the KIDSCREEN-27 quality of life questionnaire. Qual Life Res 16(8):1335–1345. https://doi.org/10.1007/s11136-007-9241-1 CrossRefPubMed

38.

Kidscreen Group Europe (2006) The KIDSCREEN questionnaires: quality of life questionnaires for children and adolescents—handbook. Pabst Science Publisher, Lengerich

39.

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 (2009) The Clavien-Dindo classification of surgical complications: five-year experience. Ann Surg 250(2):187–196. https://doi.org/10.1097/SLA.0b013e3181b13ca2 CrossRefPubMed

40.

Dindo D, Demartines N, Clavien PA (2004) Classification of surgical complications: a new proposal with evaluation in a cohort of 6336 patients and results of a survey. Ann Surg 240(2):205–213. https://doi.org/10.1097/01.sla.0000133083.54934.ae CrossRefPubMedPubMedCentral

41.

Koopmanschap MA (2005) PRODISQ: a modular questionnaire on productivity and disease for economic evaluation studies. Expert Rev Pharmacoecon Outcomes Res 5(1):23–28. https://doi.org/10.1586/14737167.5.1.23 CrossRefPubMed

42.

Zorginstituut Nederland (2016) Richtlijn voor het uitvoeren van economische evaluaties in de gezondheidszorg (Guideline for economic evaluations in health care). Zorginstituut Nederland, Diemen

43.

Zorginstituut Nederland (2016 ) Kostenhandleiding—methodologie van kostenonderzoek en referentieprijzen voor economische evaluaties in de gezondheidszorg (Guideline for costing research in economic evaluations in health care). Zorginstituut Nederland, Diemen

44.

Drost RMWA, Paulus ATG, Ruwaard D, Evers SMAA (2014) Handleiding intersectorale kosten en baten van (preventieve) interventies—classificatie, identificatie en kostprijzen (Manual intersectoral costs and benefits of (preventive) interventions - classification, identification and cost prices). Maastricht University, Maastricht

45.

Sullivan SD, Mauskopf JA, Augustovski F, Caro JJ, Lee KM, Minchin M, Olewska E, Penna P, Barrios JMR, Shau WY (2014) Principles of good practice for budget impact analysis II: report of the ISPOR Task Force on good research practices—budget impact analysis. Value Health 17:5–14. https://doi.org/10.1016/j.jval.2013.08.2291 CrossRefPubMed

46.

Knowles CH, Thin N, Gill K, Bhan C, Grimmer K, Lunniss PJ, Williams NS, Scott SM (2012) Prospective randomized double-blind study of temporary sacral nerve stimulation in patients with rectal evacuatory dysfunction and rectal hyposensitivity. Ann Surg 255(4):643–649. https://doi.org/10.1097/SLA.0b013e318247d49f CrossRefPubMed

47.

Kenefick NJ, Vaizey CJ, Cohen RG, Nicholls RJ, Kamm MA (2002) Double-blind placebo-controlled crossover study of sacral nerve stimulation for idiopathic constipation. Br J Surg 89:1570–1571CrossRefPubMed

48.

Dinning PG, Hunt L, Patton V, Zhang T, Szczesniak M, Gebski V, Jones M, Stewart P, Lubowski DZ, Cook IJ (2015) Treatment efficacy of sacral nerve stimulation in slow transit constipation: a two-phase, double-blind randomized controlled crossover study. Am J Gastroenterol 110(5):733–740. https://doi.org/10.1038/ajg.2015.101 CrossRefPubMed

49.

van der Wilt AA, van Wunnik BPW, Sturkenboom R, Han-Geurts IJ, Melenhorst J, Benninga MA, Baeten CGMI, Breukink SO (2016) Sacral neuromodulation in children and adolescents with chronic constipation refractory to conservative treatment. Int J Color Dis 31(8):1459–1466. https://doi.org/10.1007/s00384-016-2604-8 CrossRef

50.

van Wunnik BPW, Peeters B, Govaert B, Nieman FH, Benninga MA, Beaten CGMI (2012) Sacral neuromodulation therapy: a promising treatment for adolescents with refractory functional constipation. Dis Colon rectum 55:278–285. https://doi.org/10.1097/DCR.0b013e3182405c61

51.

Dinning PG, Fuentealba SE, Kennedy ML, Lobowski DZ, Cook IJ (2006) Sacral nerve stimulation induces pan-colonic propagating pressure waves and increases defecation frequency in patients with slow-transit constipation. Color Dis 9:123–132. https://doi.org/10.1111/j.1463-1318.2006.01096.x CrossRef

52.

Dinning PG, Hunt LM, Arkwright JW, Patton V, Szczesniak MM, Wiklendt L, Davidson JB, Lubowski DZ, cook IJ (2012) Pancolonic motor response to subsensory and suprasensory sacral nerve stimulation in patients with slow-transit constipation. Br J Surg 99:1002–1010. doi:https://doi.org/10.1002/bjs.8760

Title: Sacral neuromodulation versus personalized conservative treatment in patients with idiopathic slow-transit constipation: study protocol of the No.2-trial, a multicenter open-label randomized controlled trial and cost-effectiveness analysis
Authors: S. C. M. Heemskerk
A. H. Rotteveel
M. A. Benninga
C. I. M. Baeten
A. A. M. Masclee
J. Melenhorst
S. M. J. van Kuijk
C. D. Dirksen
S. O. Breukink
Publication date: 01-04-2018
Publisher: Springer Berlin Heidelberg
Published in: International Journal of Colorectal Disease / Issue 4/2018
Print ISSN: 0179-1958
Electronic ISSN: 1432-1262
DOI: https://doi.org/10.1007/s00384-018-2978-x

At a glance: The ONWARDS insulin icodec trials

Springer Medicine

Sacral neuromodulation versus personalized conservative treatment in patients with idiopathic slow-transit constipation: study protocol of the No.2-trial, a multicenter open-label randomized controlled trial and cost-effectiveness analysis

Abstract

Purpose

Methods

Conclusions

Trial registration

At a glance: The ONWARDS insulin icodec trials

Springer Medicine

Abstract

Purpose

Methods

Conclusions

Trial registration

Please log in to get access to this content

Other articles of this Issue 4/2018

New perspectives on the long-term outcome of segmental colectomy for Crohn’s colitis: an observational study on 200 patients

Bacterial culture and antibiotic susceptibility in patients with acute appendicitis

Selective association of nonaspirin NSAIDs with risk of diverticulitis

Trends in the treatment of rectal prolapse: a population analysis

Fecal incontinence after transanal endoscopic microsurgery

Screening in spouses of colorectal cancer patients: a missed opportunity