EFFICACY OF HOME-BASED INTENSIVE BIMANUAL TRAINING FOR CHILDREN WITH UNILATERAL SPASTIC CEREBRAL PALSY Claudio Luis Ferre Submitted in partial fulfillment of the requirements for the degree of Doctor of Philosophy under the Executive Committee of the Graduate School of Arts and Sciences COLUMBIA UNIVERSITY 2015 © 2015 Claudio Luis Ferre All rights reserved ABSTRACT EFFICACY OF HOME-BASED INTENSIVE BIMANUAL TRAINING FOR CHILDREN WITH UNILATERAL SPASTIC CEREBRAL PALSY Claudio Luis Ferre Neuroplasticity research suggests intervention at early developmental stages is optimal for maximizing recovery of function in children with unilateral spastic cerebral palsy (USCP). Intensive bimanual training is an effective method for improving upper- extremity function in children with USCP when provided in massed-practice day camps. Given the challenges young children face sustaining attention and their susceptibility to fatigue, adapted models using distributed practice are required. The aim of this study was to perform a randomized trial comparing home-based hand-arm bimanual intensive training (H-HABIT) with a control group receiving an intervention of equal duration, intensity, and social interaction. Twenty-four children with USCP (age range 2 yr., 6 mos. - 10 yr. 1 mos.) were randomized to participate in either 90 hours of H-HABIT (n=12) or an equivalent dose of functional lower-limb training (FLL-control; n=12). Caregivers were trained by experienced interventionists to administer either H-HABIT or FLL-control. Caregivers then performed activities with children in their own home 2 hrs./day, 5 days/week for 9 weeks (90 hrs. total). Caregivers were supervised remotely once a week for one hour using telerehabilitation. Dexterity was assessed using the Box and Blocks test. Bimanual hand function was measured using the Assisting Hand Assessment (AHA). Canadian Occupational Performance Measure (COPM) was used to assess caregiver perception (performance and satisfaction) of functional goals. All measures were assessed immediately prior to (pretest) and immediately after (posttest) the intervention and analyzed with a 2(group) x 2(session) repeated measures ANOVA. There were no statistical differences between the two groups at baseline. Children in H-HABIT showed greater improvement (pretest=9.0±5.8; posttest=14.5±7.8) than children in FLL-control (pretest=10.6±7.2; posttest=11.9±6.1) on the Box and Blocks test (for interaction, F(1,20)=18.53, p<.001). Neither group demonstrated change on the AHA (F(1,22)=0.89, p>.05) (H- HABIT=60.5 AHA units±10.1; FLL-Control=52.8 AHA units±17.1). COPM data revealed a significant test session by group interaction (F(1,22)=10.82, p<.01) with caregivers of children in H-HABIT rating higher goal performance (pretest=2.9±1.0; posttest=6.8±1.3) relative to FLL-control (pretest=2.7±1.0; posttest=4.5±1.7). Caregivers in both groups showed equal improvement between the two sessions (F(1,22)=115.63, p<.001) in ratings of satisfaction of goal performance (pretest=3.8±1.8; posttest=7.3±1.1 for H-HABIT and pretest=2.3±1.0; posttest=4.7±1.8 for FLL-control). Children in H-HABIT made greater improvements in dexterity and parent-rated goal performance. This is the first randomized trial to examine the efficacy of intensive bimanual training with caregivers as interventionists—a model which permits intervention at younger ages when there may be greater potential for improving hand function. Using caregivers as a way to implement intensive interventions provides a cost-effective alternative to expensive clinic-based interventions. Home-based models provide a valuable intervention approach to add to the repertoire of options clinicians have to chose from when developing individualized treatment programs for children and their families. TABLE OF CONTENTS CHAPTER List of Tables ............................................................................................................... iii List of Figures .............................................................................................................. iv I. INTRODUCTION ............................................................................................. 1 II. METHODS ..................................................................................................... 10 i. Participants ............................................................................................ 10 ii. Randomization ...................................................................................... 11 iii. Study Design ......................................................................................... 11 iv. Intervention Protocols ........................................................................... 12 v. Caregiver Training ................................................................................ 14 vi. Participant Classification ....................................................................... 18 vii. Primary and Secondary Outcome Measures ......................................... 19 viii. Sample Size Calculation ........................................................................ 22 ix. Statistical Analysis ................................................................................ 23 III. RESULTS ........................................................................................................ 23 i. Patient Flow ......................................................................................... 23 ii. Treatment Characteristics ................................................................... 28 iii. Consistency Between Caregiver-and Supervisor-directed Assessments ............................................................................................................. 33 iv. Dexterity .............................................................................................. 33 v. Quality of Spontaneously Using Affected Hand as an Assisting Hand ............................................................................................................. 34 vi. Caregiver-rated Functional ................................................................ .37 vii. Predictors of improvement ................................................................... 41 IV. DISCUSSION .................................................................................................. 43 i Home-based Interventions as a tool for Intensive Rehabilitation ... 43 ii Home-based Programs are Feasible, but for whom? ....................... 45 iii Feasibility of Lower-limb Training as a Control Group ................. 48 iv Children in H-HABIT Make Unimanual but not Bimanual Improvements .................................................................................. 49 i v Does Participation in an Intervention Increase Parental Satisfaction? ......................................................................................................... 52 vi Limitations and Future Considerations ........................................... 53 REFERENCES ............................................................................................................ 58 Appendix A .................................................................................................................. 66 Appendix B .................................................................................................................. 90 Appendix C .................................................................................................................. 96 Appendix D ................................................................................................................ 111 Appendix E ................................................................................................................ 121 Appendix F ................................................................................................................ 132 Appendix G ................................................................................................................ 139 Appendix H ................................................................................................................ 143 Appendix I ................................................................................................................. 146 Appendix J ................................................................................................................. 154 ii LIST OF TABLES I. Participant Characteristics ...................................................................... 27 II. Responses to Daily Log Feasibility ........................................................ 31 III. Categorization of Activities Performed During H-HABIT .................... 32 IV. Results of Hand Function Assessments by Testing Session ................... 35 V. Functional Goals Identified by Caregivers ............................................. 38 iii LIST OF FIGURES I. Patient Flow ............................................................................................ 26 II. Box and Blocks by Testing Session ........................................................ 36 III. COPM-Performance by Testing Session ................................................ 39 IV. COPM-Satisfaction by Testing Session .................................................. 40 V. Relationship Between Change in AHA and ........................................... 42 iv DEDICATION Dedicado a mis viejos. Gracias Papá por enseñarme que “hay que meterle duro” y gracias Mamá por enseñarme que “no se puede esperar que se abran las puertas, hay que patearlas abiertas”. v ACKNOWLEDGMENTS I would like to express my sincerest gratitude to the following people: Dr. Andy Gordon, my advisor, for his advice and support, and for helping me to grow as a research scientist. Thank you for teaching me how to keep a sense of humor through it all. Dr. Sunil Agrawal, Dr. Kathleen Friel, Dr. Stephen Silverman, and Dr. Carol Garber for their patience and agreeing to serve as members of my committee. Dr. Garber, the encouragement you provided when stopping by the office was invaluable. Dr. Malandraki, for the helpful feedback during the proposal stage of my project. Dr. Marina Brandão, for your Brazilian enthusiasm and for the amount of work and care you put into the project. Dr. George Michel, my master’s thesis advisor, for providing me with the foundation of the skills needed to pursue research. Dr. Jeremy Bailoo, Dr. Amber Tyler, Cherie Kuo, Celine Craje, and all my labmates, for the laughs, encouragement, and therapeutic happy hours. Dr. Noelle Moreau, Bhavini Surana, and Ashley Dew, for doing such a fantastic job with the lower-limb group. My family, Eddie, Giovanna, and Gian-Alexi, for the encouragement and love. Thank you to my mother and father for nurturing my curiosity with their unwavering support and constant belief in me. Masha, for being Masha. Rachel, for her love, understanding, and daily smiles. vi