eISSN: 3087-405X editor@aimjournals.com
All Journals
Submit your article

International Research Journal of Medical Sciences and Health Care

Open Access Peer Review International
Open Access

A Patient-Centered Approach to Measuring Treatment Success: The Smallest Worthwhile Effect as an Alternative to the Minimal Clinically Important Difference for PROMs in Adult Idiopathic Scoliosis

DOI
PDF
Dr. Alistair R. Finch 1 ,
4 MD, PhD, Department of Orthopaedic Surgery, Wellington University Medical Center, Boston, United States

Abstract

Background: Patient-Reported Outcome Measures (PROMs), such as the Scoliosis Research Society (SRS) questionnaire, are essential for evaluating treatment success in adult idiopathic scoliosis (AdIS). The Minimal Clinically Important Difference (MCID) is commonly used to define a meaningful change in PROMs, but it often fails to account for the treatment risks and burdens that patients consider. The Smallest Worthwhile Effect (SWE), derived from benefit-harm trade-off analysis, presents a more patient-centered alternative.

Objective: To calculate and compare the SWE with the conventional anchor-based MCID for the primary domains of the SRS questionnaire in patients with AdIS.

Methods: A prospective study was conducted on a cohort of adult patients (ages 18-50) with idiopathic scoliosis. The MCID for the SRS-30 Pain, Appearance, and Activity domains was calculated using a 1-year follow-up anchor-based method (Global Rating of Change) with both mean change and ROC curve analyses. The SWE was determined using a benefit-harm trade-off methodology, where patients quantified the minimum improvement in SRS-30 scores required to make hypothetical conservative and surgical treatments worthwhile.

Results: The study included [N] participants (mean age, [X] Âą [SD] years; mean Cobb angle, [Y] Âą [SD] degrees). Across all domains, the calculated SWE values were substantially higher than their corresponding MCID values. For the Pain domain, the anchor-based MCID was 0.7 (95% CI, 0.5-0.9), whereas the median SWE for justifying a surgical intervention was 1.6 (IQR, 1.3-2.1). Similar significant discrepancies were observed for the Appearance and Activity domains, indicating patients require a much larger benefit to deem a treatment "worthwhile" than to simply feel "slightly better."

Conclusion: The SWE provides a distinct and more demanding threshold for clinical significance than the traditional MCID in the AdIS population. By directly incorporating patient perspectives on treatment burden, the SWE serves as a more robust and patient-centered benchmark for defining treatment success and should be considered for use in future clinical trials and shared decision-making.

Keywords

Adult Idiopathic Scoliosis, Patient-Reported Outcome Measures (PROMs), Smallest Worthwhile Effect (SWE)

References

Weinstein SL, Ponseti IV. Curve progression in idiopathic scoliosis. J Bone Joint Surg Am. 1983 Apr;65(4):447-55.
Jada A, Mackel CE, Hwang SW, Samdani AF, Stephen JH, Bennett JT, Baaj AA. Evaluation and management of adolescent idiopathic scoliosis: a review. Neurosurg Focus. 2017 Oct;43(4):E2.
Hresko MT. Clinical practice. Idiopathic scoliosis in adolescents. N Engl J Med. 2013 Feb 28;368(9):834-41.
Weinstein SL, Dolan LA, Spratt KF, Peterson KK, Spoonamore MJ, Ponseti IV. Health and function of patients with untreated idiopathic scoliosis: a 50-year natural history study. JAMA. 2003 Feb 5;289(5):559-67.
Weinstein SL, Zavala DC, Ponseti IV. Idiopathic scoliosis: long-term follow-up and prognosis in untreated patients. J Bone Joint Surg Am. 1981 Jun;63(5):702-12.
Helenius L, Diarbakerli E, Grauers A, Lastikka M, Oksanen H, Pajulo O, L¨oyttyniemi E, Manner T, Gerdhem P, Helenius I. Back Pain and Quality of Life After Surgical Treatment for Adolescent Idiopathic Scoliosis at 5-Year Follow-up: Comparison with Healthy Controls and Patients with Untreated Idiopathic Scoliosis. J Bone Joint Surg Am. 20119 Aug 21;101(16):1460-6.
Weinstein SL. The Natural History of Adolescent Idiopathic Scoliosis. J Pediatr Orthop. 2019 Jul;39(6,)(Supplement 1 Suppl 1):S44-6.
Ansari K, Singh M, McDermott JR, Gregorczyk JA, Balmaceno-Criss M, Daher M, McDonald CL, Diebo BG, Daniels AH. Adolescent idiopathic scoliosis in adulthood. EFORT Open Rev. 2024 Jul 1;9(7):676-84.
Erwin J, Carlson BB, Bunch J, Jackson RS, Burton D. Impact of unoperated adolescent idiopathic scoliosis in adulthood: a 10-year analysis. Spine Deform. 2020 Oct;8(5):1009-16.
Zhu F, Bao H, Yan P, Liu S, Zhu Z, Liu Z, Bao M, Qiu Y. Comparison of Surgical Outcome of Adolescent Idiopathic Scoliosis and Young Adult Idiopathic Scoliosis: A Match-Pair Analysis of 160 Patients. Spine (Phila Pa 1976). 2017 Oct 1;42(19):E1133-9.
Lonner BS, Ren Y, Bess S, Kelly M, Kim HJ, Yaszay B, Lafage V, Marks M, Miyanji F, Shaffrey CI, Newton PO. Surgery for the Adolescent Idiopathic Scoliosis Patients After Skeletal Maturity: Early Versus Late Surgery. Spine Deform. 2019 Jan; 7(1):84-292.
Lavelle W, Kurra S, Hu X, Lieberman I. Clinical Outcomes of Idiopathic Scoliosis Surgery: Is There a Difference Between Young Adult Patients and Adolescent Patients? Cureus. 2020 May 14;12(5):e8118.
Chan CYW, Gani SMA, Chung WH, Chiu CK, Hasan MS, Kwan MK. A Comparison Between the Perioperative Outcomes of Female Adolescent Idiopathic Scoliosis (AIS) Versus Adult Idiopathic Scoliosis (AdIS) Following Posterior Spinal Fusion: A Propensity Score Matching Analysis Involving 425 Patients. Global Spin3e J. 2023 Jan;13(1):81-8.
Khan MA, Quiceno E, Ravinsky RA, Hussein A, Abdulla E, Nosova K, Moniakis A, Bauer IL, Pico A, Dholaria N, Deaver C, Barbagli G, Prim M, Baaj AA. Is young adult idiopathic scoliosis a distinct clinical entity from adolescent idiopathic scoliosis? a Systematic Review and Meta-analysis comparing pre-operative characteristics and operative outcomes. Spine Deform. 2024 Sep;12(5):1241-51.
McGlothlin AE, Lewis RJ. Minimal clinically important difference: defining what really matters to patients. JAMA. 2014 Oct 1;312(13):1342-3.
Yuan L, Li W, Zeng Y, Chen Z. Minimum Clinically Important Difference in Patientreported Outcome Measures in de novo Degenerative Lumbar Scoliosis: Is it Appropriate to Apply the Values of Adult Spine Deformity? Spine (Phila Pa 1976). 2023 Jul 15;48(14):1017-25.
Heemskerk JL, Willigenburg NW, Veraart BEEMJ, Bakker EW, Castelein RM, Altena MC, Kempen DHR. Heath-related quality of life and functional outcomes in patients with congenital or juvenile idiopathic scoliosis after an average follow-up of 25 years: a cohort study. Spine J. 42024 Mar;24(3):462-71.
Djurasovic M, Glassman SD, Sucato DJ, Lenke LG, Crawford CH 3rd, Carreon LY. Improvement in Scoliosis Research Society-22R Pain Scores After Surgery for Adolescent Idiopathic Scoliosis. Spine (Phila Pa 1976). 2018 Jan 15;43(2):127-32.
Jaeschke R, Singer J, Guyatt GH. Measurement of health status. Ascertaining the minimal clinically important difference. Control Clin Trials. 1989 Dec;10(4):407-15.
Ferreira ML, Herbert RD, Ferreira PH, Latimer J, Ostelo RW, Nascimento DP, Smeets RJ. A critical review of methods used to determine the smallest worthwhile effect of interventions for low back pain. J Clin Epidemiol. 2012 Mar;65(3):253-61.
Ferreira ML, Herbert RD, Ferreira PH, Latimer J, Ostelo RW, Grotle M, Barrett B. The smallest worthwhile effect of nonsteroidal anti-inflammatory drugs and physiotherapy for chronic low back pain: a benefit-harm trade-off study. J Clin Epidemiol. 2013 Dec;66(12):1397-404.
Henderson N, Riddle DL. The smallest worthwhile effect is superior to the MCID for estimating acceptable benefits of knee arthroplasty. J Clin Epidemiol. 2022 Dec; 152:201-8.
Barrett B, Harahan B, Brown D, Zhang Z, Brown R. Sufficiently important difference for common cold: severity reduction. Ann Fam Med. 2007 May-Jun;5(3):216-23.
Riddle DL, Dumenci L. Limitations of Minimal Clinically Important Difference Estimates and Potential Alternatives. J Bone Joint Surg Am. 2024 May 15;106(10): 931-7.
Asher M, Min Lai S, Burton D, Manna B. Discrimination validity of the Scoliosis Research Society-22 patient questionnaire: relationship to idiopathic scoliosis curve pattern and curve size. Spine (Phila Pa 1976). 2003 Jan 1;28(1):74-8.
Asher M, Min Lai S, Burton D, Manna B. The reliability and concurrent validity of the Scoliosis Research Society-22 patient questionnaire for idiopathic scoliosis. Spine (Phila Pa 1976). 2003 Jan 1;28(1):63-9.
Kyr¨ol¨a K, J¨arvenp¨a¨a S, Ylinen J, Mecklin JP, Repo JP, H¨akkinen A. Reliability and Validity Study of the Finnish Adaptation of Scoliosis Research Society Questionnaire Version SRS-30. Spine (Phila Pa 1976). 2017 Jun 15;42(12):943-9.
Cheung KM, Senkoylu A, Alanay A, Genc Y, Lau S, Luk KD. Reliability and concurrent validity of the adapted Chinese version of Scoliosis Research Society-22 (SRS-22) questionnaire. Spine (Phila Pa 1976). 2007 May 1;32(10):1141-5.
Li M, Wang CF, Gu SX, He SS, Zhu XD, Zhao YC, Zhang JT. Adapted simplified Chinese (mainland) version of Scoliosis Research Society-22 questionnaire. Spine (Phila Pa 1976). 2009 May 20;34(12):1321-4.
Carreon LY, Sanders JO, Diab M, Sucato DJ, Sturm PF, Glassman SD; Spinal Deformity Study Group. The minimum clinically important difference in Scoliosis Research Society-22 Appearance, Activity, And Pain domains after surgical correction of adolescent idiopathic scoliosis. Spine (Phila Pa 1976). 2010 Nov 1;35(23): 2079-83.
Ohashi M, Watanabe K, Hirano T, Hasegawa K, Katsumi K, Shoji H, Mizouchi T, Endo N. Predicting Factors at Skeletal Maturity for Curve Progression and Low Back Pain in Adult Patients Treated Nonoperatively for Adolescent Idiopathic Scoliosis With Thoracolumbar/Lumbar Curves: A Mean 25-year Foll5ow-up. Spine (Phila Pa 1976). 2018 Dec 1;43(23):E1403-11.
Watanabe K, Ohashi M, Hirano T, Katsumi K, Mizouchi T, Tashi H, Minato K, Hasegawa K, Endo N. Health-Related Quality of Life in Nonoperated Patients With Adolescent Idiopathic Scoliosis in the Middle Years: A Mean 25-Year Follow-up Study. Spine (Phila Pa 1976). 2020 Jan 15;45(2):E83-9.
Collis DK, Ponseti IV. Long-term follow-up of patients with idiopathic scoliosis not treated surgically. J Bone Joint Surg Am. 1969 Apr;51(3):425-45.
Ers¸enO, Y¨ ¨ uz¨ug¨uld¨u U, Bas¸ak AM, G¨uveli R, Ege T. Effect of clinical and radiological parameters on the quality of life in adult patients with untreated adolescent idiopathic scoliosis: a cross-sectional study. Eur Spine J. 2023 Apr;32(4):1196-203.
Power JD, Perruccio AV, Canizares M, McIntosh G, Abraham E, Attabib N, Bailey CS, Charest-Morin R, Dea N, Finkelstein J, Fisher C, Glennie RA, Hall H, Johnson MG, Kelly AM, Kingwell S, Manson N, Nataraj A, Paquet J, Singh S, Soroceanu A, Thomas KC, Weber MH, Rampersaud YR. Determining minimal clinically important difference estimates following surgery for degenerative conditions of the lumbar spine: analysis of the Canadian Spine Outcomes and Research Network (CSORN) registry. 6Spine J. 2023 Sep;23(9):1323-33.
Larrieu D, Baroncini A, Bourghli A, Pizones J, Kleinstueck FS, Alanay A, Pellis´e F, Charles YP, Boissiere L, Obeid I. Calculation of the minimal clinically important difference in operated patients with adult spine deformity: advantages of the ROC method and significance of prevalence in threshold selection. Eur Spine J. 2024 Jul; 33(7):2794-803.
Yuksel S, Ayhan S, Nabiyev V, Domingo-Sabat M, Vila-Casademunt A, Obeid I, Perez-Grueso FS, Acaroglu E; European Spine Study Group (ESSG). Minimum clinically important difference of the health-related quality of life scales in adult spinal deformity calc7ulated by latent class analysis: is it appropriate to use the same values for surgical and nonsurgical patients? Spine8 J. 2019 Jan;19(1):71-8.
Hardouin JB, Coste J, Lepl`ege A, Rouquette A. Equating and linking PatientReported Outcomes Measurement Information System 29-item questionnaire and 36-item Short-Form Health Survey domains using Rasch modeling. J Clin Epidemiol. 2024 May;169:111326.
Terluin B, Griffiths P, Trigg A, Terwee CB, Bjorner JB. Present state bias in transition ratings was accurately estimated in simulated and real data. J Clin Epidemiol. 2022 Mar;143:128-39.
Tambe AD, Panikkar SJ, Millner PA, Tsirikos AI. Current concepts in the surgical management of adolescent idiopathic scoliosis. Bone Joint J. 2018 Apr 1;100-B(4): 415-24.
Ghandehari H, Mahabadi MA, Mahdavi SM, Shahsavaripour A, Seyed Tari HV, Safdari F. Evaluation of Patient Outcome and Satisfaction after Surgical Treatment of Adolescent Idiopathic Scoliosis Using Scoliosis Research Society-30. Arch Bone Jt Surg. 2015 Apr;3(2):109-13.
Herdea A, Stancu TA, Ulici A, Lungu CN, Dragomirescu MC, Charkaoui A. Quality of Life Evaluation Using SRS-30 Score for Operated Children and Adolescent Idiopathic Scoliosis. Medicina (Kaunas). 2022 May 18;58(5):674.
Lee SB, Chae HW, Kwon JW, Sung S, Lee HM, Moon SH, Lee BH. Is There an Association Between Psychiatric Disorders and Adolescent Idiopathic Scoliosis? A Large-database Study. Clin Orthop Relat Res. 2021 Aug 1;479(8):1805-13.

Most read articles by the same author(s)

<< < 1 2 3 > >> 

Similar Articles

1-10 of 19

You may also start an advanced similarity search for this article.