Assessment of proxy-reported responses as predictors of motor and sensory peripheral neuropathy in children with B-lymphoblastic leukemia
Corresponding Author
Rozalyn L. Rodwin
Department of Pediatrics, Yale School of Medicine, New Haven, Connecticut, USA
Yale Cancer Center, New Haven, Connecticut, USA
Correspondence
Rozalyn. L Rodwin, Section of Pediatric Hematology/Oncology, Deparatment of Pediatrics, Yale School of Medicine, 330 Cedar Street, LMP 2073, PO Box 208064, New Haven, CT, 06520.
Email: [email protected]
Search for more papers by this authorNatalie J. DelRocco
Department of Biostatistics, Colleges of Medicine and Public Health & Health Professions, University of Florida, Gainesville, Florida, USA
Search for more papers by this authorEmily Hibbitts
Department of Biostatistics, Colleges of Medicine and Public Health & Health Professions, University of Florida, Gainesville, Florida, USA
Search for more papers by this authorMeenakshi Devidas
Department of Global Pediatric Medicine, St. Jude Children's Research Hospital, Memphis, Tennessee, USA
Search for more papers by this authorMoira K. Whitley
Department of Pediatrics, Yale School of Medicine, New Haven, Connecticut, USA
Search for more papers by this authorCaroline E. Mohrmann
Department of Pediatrics, Washington University School of Medicine, St. Louis Children's Hospital, St. Louis, Missouri, USA
Goldfarb School of Nursing, St. Louis, Missouri, USA
Search for more papers by this authorReuven J. Schore
Center of Cancer and Blood Disorders, Children's National Health System, Washington, District of Columbia, USA
George Washington University School of Medicine and Health Sciences, Washington, District of Columbia, USA
Search for more papers by this authorElizabeth Raetz
Department of Pediatrics, NYU Langone Health, New York, New York, USA
Search for more papers by this authorNaomi J. Winick
Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, Texas, USA
Search for more papers by this authorStephen P. Hunger
Department of Pediatrics and the Center for Childhood Cancer Research, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA
Search for more papers by this authorMignon L. Loh
Division of Pediatric Hematology, Oncology, Bone Marrow Transplant and Cellular Therapy, Seattle Children's Hospital and the Ben Towne Center for Childhood Cancer Research, University of Washington, Seattle, Washington, USA
Search for more papers by this authorMarilyn J. Hockenberry
Department of Pediatrics, Baylor College of Medicine, Houston, Texas, USA
School of Nursing, Duke University, Durham, North Carolina, USA
Search for more papers by this authorXiaomei Ma
Yale Cancer Center, New Haven, Connecticut, USA
Department of Chronic Disease Epidemiology, Yale School of Public Health, New Haven, Connecticut, USA
Cancer Outcomes, Public Policy, and Effectiveness Research (COPPER) Center, Yale University, New Haven, Connecticut, USA
Search for more papers by this authorAnne L. Angiolillo
Center of Cancer and Blood Disorders, Children's National Health System, Washington, District of Columbia, USA
George Washington University School of Medicine and Health Sciences, Washington, District of Columbia, USA
Servier Pharmaceuticals, Boston, Massachusetts, USA
Search for more papers by this authorKirsten K. Ness
Department of Epidemiology and Cancer Control, St. Jude Children's Research Hospital, Memphis, Tennessee, USA
Search for more papers by this authorJohn A. Kairalla
Department of Biostatistics, Colleges of Medicine and Public Health & Health Professions, University of Florida, Gainesville, Florida, USA
Search for more papers by this authorNina S. Kadan-Lottick
Cancer Prevention and Control Program, Georgetown Lombardi Comprehensive Cancer Center, Washington, District of Columbia, USA
Search for more papers by this authorCorresponding Author
Rozalyn L. Rodwin
Department of Pediatrics, Yale School of Medicine, New Haven, Connecticut, USA
Yale Cancer Center, New Haven, Connecticut, USA
Correspondence
Rozalyn. L Rodwin, Section of Pediatric Hematology/Oncology, Deparatment of Pediatrics, Yale School of Medicine, 330 Cedar Street, LMP 2073, PO Box 208064, New Haven, CT, 06520.
Email: [email protected]
Search for more papers by this authorNatalie J. DelRocco
Department of Biostatistics, Colleges of Medicine and Public Health & Health Professions, University of Florida, Gainesville, Florida, USA
Search for more papers by this authorEmily Hibbitts
Department of Biostatistics, Colleges of Medicine and Public Health & Health Professions, University of Florida, Gainesville, Florida, USA
Search for more papers by this authorMeenakshi Devidas
Department of Global Pediatric Medicine, St. Jude Children's Research Hospital, Memphis, Tennessee, USA
Search for more papers by this authorMoira K. Whitley
Department of Pediatrics, Yale School of Medicine, New Haven, Connecticut, USA
Search for more papers by this authorCaroline E. Mohrmann
Department of Pediatrics, Washington University School of Medicine, St. Louis Children's Hospital, St. Louis, Missouri, USA
Goldfarb School of Nursing, St. Louis, Missouri, USA
Search for more papers by this authorReuven J. Schore
Center of Cancer and Blood Disorders, Children's National Health System, Washington, District of Columbia, USA
George Washington University School of Medicine and Health Sciences, Washington, District of Columbia, USA
Search for more papers by this authorElizabeth Raetz
Department of Pediatrics, NYU Langone Health, New York, New York, USA
Search for more papers by this authorNaomi J. Winick
Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, Texas, USA
Search for more papers by this authorStephen P. Hunger
Department of Pediatrics and the Center for Childhood Cancer Research, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA
Search for more papers by this authorMignon L. Loh
Division of Pediatric Hematology, Oncology, Bone Marrow Transplant and Cellular Therapy, Seattle Children's Hospital and the Ben Towne Center for Childhood Cancer Research, University of Washington, Seattle, Washington, USA
Search for more papers by this authorMarilyn J. Hockenberry
Department of Pediatrics, Baylor College of Medicine, Houston, Texas, USA
School of Nursing, Duke University, Durham, North Carolina, USA
Search for more papers by this authorXiaomei Ma
Yale Cancer Center, New Haven, Connecticut, USA
Department of Chronic Disease Epidemiology, Yale School of Public Health, New Haven, Connecticut, USA
Cancer Outcomes, Public Policy, and Effectiveness Research (COPPER) Center, Yale University, New Haven, Connecticut, USA
Search for more papers by this authorAnne L. Angiolillo
Center of Cancer and Blood Disorders, Children's National Health System, Washington, District of Columbia, USA
George Washington University School of Medicine and Health Sciences, Washington, District of Columbia, USA
Servier Pharmaceuticals, Boston, Massachusetts, USA
Search for more papers by this authorKirsten K. Ness
Department of Epidemiology and Cancer Control, St. Jude Children's Research Hospital, Memphis, Tennessee, USA
Search for more papers by this authorJohn A. Kairalla
Department of Biostatistics, Colleges of Medicine and Public Health & Health Professions, University of Florida, Gainesville, Florida, USA
Search for more papers by this authorNina S. Kadan-Lottick
Cancer Prevention and Control Program, Georgetown Lombardi Comprehensive Cancer Center, Washington, District of Columbia, USA
Search for more papers by this authorAbstract
Chemotherapy-induced peripheral neuropathy (CIPN), a common condition in children with acute lymphoblastic leukemia, can be challenging to diagnose. Using data from Children's Oncology Group AALL0932 physical function study, we sought to determine if parent/guardian proxy-reported responses from the Pediatric Outcomes Data Collection Instrument could identify children with motor or sensory CIPN diagnosed by physical/occupational therapists (PT/OT). Four variables moderately discriminated between children with and without motor CIPN (c-index 0.76, 95% confidence interval [CI]: 0.64–0.84), but sensory and optimism-corrected models had weak discrimination (c-index sensory models 0.65, 95% CI: 0.54–0.74). New proxy-report measures are needed to identify children with PT/OT diagnosed CIPN.
CONFLICT OF INTEREST STATEMENT
Stephen P. Hunger owns common stock in Amgen and has received honoraria from Amgen, Jazz, and Servier. Anne L. Angiolillo is employed by Servier Pharmaceuticals.
Open Research
DATA AVAILABILITY STATEMENT
De-identified data would be shared with written request to corresponding author, and would require detailed analysis plan, approval by the Children's Oncology Group Acute Lymphoblastic Leukemia Committee Chair, and data usage agreement.
Supporting Information
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pbc30634-sup-001-SuppMat.docx195.8 KB | |
pbc30634-sup-0002-FigureS1.docx77.3 KB |
Please note: The publisher is not responsible for the content or functionality of any supporting information supplied by the authors. Any queries (other than missing content) should be directed to the corresponding author for the article.
REFERENCES
- 1Kandula T, Farrar MA, Cohn RJ, et al. Chemotherapy-induced peripheral neuropathy in long-term survivors of childhood cancer: clinical, neurophysiological, functional, and patient-reported outcomes. JAMA Neurol. 2018; 75(8): 980-988. doi:10.1001/jamaneurol.2018.0963
- 2Jain P, Gulati S, Seth R, Bakhshi S, Toteja GS, Pandey RM. Vincristine-induced neuropathy in childhood ALL (acute lymphoblastic leukemia) survivors: prevalence and electrophysiological characteristics. J Child Neurol. 2014; 29(7): 932-937. doi:10.1177/0883073813491829
- 3Lavoie Smith EM, Li L, Chiang C, et al. Patterns and severity of vincristine-induced peripheral neuropathy in children with acute lymphoblastic leukemia. J Peripher Nerv Syst. 2015; 20(1): 37-46. doi:10.1111/jns.12114
- 4Tay CG, Lee VWM, Ong LC, Goh KJ, Ariffin H, Fong CY. Vincristine-induced peripheral neuropathy in survivors of childhood acute lymphoblastic leukaemia. Pediatr Blood Cancer. 2017; 64(8):e26471. doi:10.1002/pbc.26471
- 5Varedi M, Lu L, Howell CR, et al. Peripheral neuropathy, sensory processing, and balance in survivors of acute lymphoblastic leukemia. J Clin Oncol. 2018; 36(22): 2315-2322. doi:10.1200/jco.2017.76.7871
- 6Rodwin RL, Kairalla JA, Hibbitts E, et al. Persistence of chemotherapy-induced peripheral neuropathy despite vincristine reduction in childhood b-acute lymphoblastic leukemia. J Natl Cancer Inst. 2022; 114(8): 1167-1175. doi:10.1093/jnci/djac095
- 7Bjornard KL, Gilchrist LS, Inaba H, et al. Peripheral neuropathy in children and adolescents treated for cancer. Lancet Child Adolesc Health. 2018; 2(10): 744-754. doi:10.1016/s2352-4642(18)30236-0
- 8Rodwin RL, Chen Y, Yasui Y, et al. Longitudinal evaluation of neuromuscular dysfunction in long-term survivors of childhood cancer: a report from the Childhood Cancer Survivor Study. Cancer Epidemiol Biomarkers Prev. 2021; 30(8): 1536-1545. doi:10.1158/1055-9965.Epi-21-0154
- 9Fernandez-Pineda I, Hudson MM, Pappo AS, et al. Long-term functional outcomes and quality of life in adult survivors of childhood extremity sarcomas: a report from the St. Jude Lifetime Cohort Study. J Cancer Surviv. 2017; 11(1): 1-12. doi:10.1007/s11764-016-0556-1
- 10Rodwin RL, Ma X, Ness KK, Kadan-Lottick NS, Wang R. Physical therapy utilization among hospitalized patients with pediatric acute lymphoblastic leukemia. JCO Oncol Pract. 2022; 18(7): e1060-e1068. doi:10.1200/op.21.00796
- 11Tanner L, Sencer S, Hooke MC. The stoplight program: a proactive physical therapy intervention for children with acute lymphoblastic leukemia. J Pediatr Oncol Nurs. 2017; 34(5): 347-357. doi:10.1177/1043454217698093
- 12Tanner LR, Hooke MC. Improving body function and minimizing activity limitations in pediatric leukemia survivors: the lasting impact of the Stoplight Program. Pediatr Blood Cancer. 2019; 66(5):e27596. doi:10.1002/pbc.27596
- 13Rodwin RL, Ross WL, Rotatori J, et al. Newly identified chemotherapy-induced peripheral neuropathy in a childhood cancer survivorship clinic. Pediatr Blood Cancer. 2021; 69:e29550. doi:10.1002/pbc.29550
- 14Smith EML, Kuisell C, Kanzawa-Lee GA, et al. Approaches to measure paediatric chemotherapy-induced peripheral neurotoxicity: a systematic review. Lancet Haematol. 2020; 7(5): e408-e417. doi:10.1016/s2352-3026(20)30064-8
- 15Smolik S, Arland L, Hensley MA, et al. Assessment tools for peripheral neuropathy in pediatric oncology: a systematic review from the children's oncology group. J Pediatr Oncol Nurs. 2018; 35(4): 267-275. doi:10.1177/1043454218762705
- 16Gilchrist LS, Tanner L. The pediatric-modified total neuropathy score: a reliable and valid measure of chemotherapy-induced peripheral neuropathy in children with non-CNS cancers. Support Care Cancer. 2013; 21(3): 847-856. doi:10.1007/s00520-012-1591-8
- 17Lavoie Smith EM. Measuring vincristine-induced peripheral neuropathy in children with acute lymphoblastic leukemia. Cancer Nurs. 2013; 36(5): E49-E60.
- 18Smith EML, Kuisell C, Kanzawa-Lee G, et al. Assessment of pediatric chemotherapy-induced peripheral neuropathy using a new Patient-Reported outcome measure: the P-CIN. J Pediatr Oncol Nurs. 2021; 38(2): 131-141. doi:10.1177/1043454220980253
- 19 The Childhood Cancer Survivor Study public access data tables. St. Jude Children's Research Hospital. Accessed October 11, 2022. https://ccss.stjude.org/public-access-data/public-access-data-tables.html
- 20Varni JW, Seid M, Kurtin PS. PedsQL 4.0: reliability and validity of the pediatric quality of life inventory version 4.0 generic core scales in healthy and patient populations. Med Care. 2001; 39(8): 800-812. doi:10.1097/00005650-200108000-00006
- 21Daltroy LH, Liang MH, Fossel AH, Goldberg MJ. The POSNA pediatric musculoskeletal functional health questionnaire: report on reliability, validity, and sensitivity to change. Pediatric Outcomes Instrument Development Group. Pediatric Orthopaedic Society of North America. J Pediatr Orthop. 1998; 18(5): 561-571. doi:10.1097/00004694-199809000-00001
- 22Angiolillo AL, Schore RJ, Kairalla JA, et al. Excellent outcomes with reduced frequency of vincristine and dexamethasone pulses in standard-risk B-lymphoblastic leukemia: results from children's oncology group AALL0932. J Clin Oncol. 2021; 39(13): 1437-1447. doi:10.1200/jco.20.00494
- 23Blankenburg M, Boekens H, Hechler T, et al. Reference values for quantitative sensory testing in children and adolescents: developmental and gender differences of somatosensory perception. Pain. 2010; 149(1): 76-88. doi:10.1016/j.pain.2010.01.011
- 24Bohannon RW, Wang YC, Bubela D, Gershon RC. Handgrip strength: a population-based study of norms and age trajectories for 3- to 17-year-olds. Pediatr Phys Ther. 2017; 29(2): 118-123. doi:10.1097/pep.0000000000000366
- 25Beenakker EA, van der Hoeven JH, Fock JM, Maurits NM. Reference values of maximum isometric muscle force obtained in 270 children aged 4–16 years by hand-held dynamometry. Neuromuscul Disord. 2001; 11(5): 441-446. doi:10.1016/s0960-8966(01)00193-6
- 26Soucie JM, Wang C, Forsyth A, et al. Range of motion measurements: reference values and a database for comparison studies. Haemophilia. 2011; 17(3): 500-507. doi:10.1111/j.1365-2516.2010.02399.x
- 27Shotwell C, Moore ES. Assessing reliability and validity of a functional outcome measure for adolescents with hypermobility spectrum disorder. Disabil Rehabil. 2022; 44(7): 1123-1128. doi:10.1080/09638288.2020.1788177
- 28Scott KS, Barbosa GO, Pan J, Heathcock JC. Using the PODCI to measure motor function and parent expectations in children with cerebral palsy. Phys Ther. 2021; 101(12):pzab215. doi:10.1093/ptj/pzab215
- 29Amor CJ, Spaeth MC, Chafey DH, Gogola GR. Use of the pediatric outcomes data collection instrument to evaluate functional outcomes in arthrogryposis. J Pediatr Orthop. 2011; 31(3): 293-296. doi:10.1097/BPO.0b013e31820cad93
- 30Johnson BA, Sheng X, Perry AS, Stevenson DA. Activity and participation in children with neurofibromatosis type 1. Res Dev Disabil. 2015; 36: 213-221. doi:10.1016/j.ridd.2014.10.004
- 31Podeszwa DA, Stanko KJ, Mooney JF 3rd, Cramer KE, Mendelow MJ. An analysis of the functional health of obese children and adolescents utilizing the PODC instrument. J Pediatr Orthop. 2006; 26(1): 140-143. doi:10.1097/01.bpo.0000187992.09763.db
- 32Murali CN, Cuthbertson D, Slater B, et al. Pediatric outcomes data collection instrument is a useful patient-reported outcome measure for physical function in children with osteogenesis imperfecta. Genet Med. 2020; 22(3): 581-589. doi:10.1038/s41436-019-0688-6
- 33Sundin Palmeira de Oliveira N, Rocha Dias da Silva M, Bedeschi Rego de Mattos C. Use of pediatric outcomes data collection instrument to evaluate functional outcomes in multiple hereditary exostoses. J Pediatr Orthop. 2023; 43(5): 332-336. doi:10.1097/bpo.0000000000002372
- 34Pakulis PJ, Young NL, Davis AM. Evaluating physical function in an adolescent bone tumor population. Pediatr Blood Cancer. 2005; 45(5): 635-643. doi:10.1002/pbc.20383
- 35Helito AC, Lindoso L, Sieczkowska SM, et al. Poor sleep quality and health-related quality of life impact in adolescents with and without chronic immunosuppressive conditions during COVID-19 quarantine. Clinics (Sao Paulo). 2021; 76:e3501. doi:10.6061/clinics/2021/e3501
- 36Steyerberg EW, Harrell FE Jr, Borsboom GJ, Eijkemans MJ, Vergouwe Y, Habbema JD. Internal validation of predictive models: efficiency of some procedures for logistic regression analysis. J Clin Epidemiol. 2001; 54(8): 774-781. doi:10.1016/s0895-4356(01)00341-9
- 37Heinze G, Wallisch C, Dunkler D. Variable selection—a review and recommendations for the practicing statistician. Biom J. 2018; 60(3): 431-449. doi:10.1002/bimj.201700067
- 38Buuren SV, Groothuis-Oudshoorn K. Multivariate imputation by chained equations in R. J Stat Softw. 2011; 45(3): 1-67. doi:10.18637/jss.v045.i03
- 39 psfmi: Prediction Model Pooling, Selection and Performance Evaluation Across Multiply Imputed Datasets. R package version 1.0.0. R-project; 2021. Accessed May 25, 2023 https://CRAN.R-project.org/package=psfmi
- 40McLernon DJ, Giardiello D, Van Calster B, et al. Assessing performance and clinical usefulness in prediction models with survival outcomes: practical guidance for Cox proportional hazards models. Ann Intern Med. 2023; 176(1): 105-114. doi:10.7326/m22-0844
- 41Lavoie SEM, Li L, Hutchinson RJ, et al. Measuring vincristine-induced peripheral neuropathy in children with acute lymphoblastic leukemia. Cancer Nurs. 2013; 36(5): E49-E60. doi:10.1097/NCC.0b013e318299ad23
- 42Gilchrist LS, Marais L, Tanner L. Comparison of two chemotherapy-induced peripheral neuropathy measurement approaches in children. Support Care Cancer. 2014; 22(2): 359-366. doi:10.1007/s00520-013-1981-6
- 43Smith EML, Kuisell C, Cho Y, et al. Characteristics and patterns of pediatric chemotherapy-induced peripheral neuropathy: a systematic review. Cancer Treat Res Commun. 2021; 28:100420. doi:10.1016/j.ctarc.2021.100420
- 44Gilchrist LS, Tanner LR, Ness KK. Short-term recovery of chemotherapy-induced peripheral neuropathy after treatment for pediatric non-CNS cancer. Pediatr Blood Cancer. 2017; 64(1): 180-187. doi:10.1002/pbc.26204
- 45Gilchrist L, Tanner L. Gait patterns in children with cancer and vincristine neuropathy. Pediatr Phys Ther. 2016; 28(1): 16-22. doi:10.1097/pep.0000000000000208
- 46Riley RD, Snell KI, Ensor J, et al. Minimum sample size for developing a multivariable prediction model: part II—binary and time-to-event outcomes. Stat Med. 2019; 38(7): 1276-1296. doi:10.1002/sim.7992
- 47Lynn MR. Determination and quantification of content validity. Nurs Res. 1986; 35(6): 382-385.