Sickle Cell Disease
Conditions
Keywords
Pediatrics, Cognitive behavioral therapy, Behavioral intervention
Brief summary
The purpose of this study is to find out how teenagers with chronic pain and sickle cell disease respond to a new training program called Back2Life and get their feedback about how to modify the program to best fit their needs. The Back2Life training program focuses on teaching pain coping skills (also known as cognitive-behavioral therapy). The program teaches skills and strategies that may help teens improve chronic pain management and get back into their everyday activities.
Detailed description
Sickle cell disease (SCD) is a genetic disorder of the hemoglobin in which the course of acute pain from vaso-occlusion and its sequelae vary widely across genotypes and individual patients. SCD pain often begins during childhood and can progress to chronic pain for approximately 23% of children and adolescents. Youth with chronic SCD pain, that is pain that is present on most days per month and persists for at least 6 months, report high levels of functional disability, elevated depressive and anxiety symptoms, and reduced quality of life relative to youth with SCD without chronic pain. The complex, multifactorial nature of chronic SCD pain can also contribute to increased healthcare utilization for pain. The most effective management and treatment of chronic SCD pain likely requires individualized, multimodal, multidisciplinary treatments that go beyond pharmacological management alone. A range of evidence-based non-pharmacological treatments, such as behavioral health, complementary, and integrative health approaches, are recommended for chronic pain management and are gaining greater awareness and integration into comprehensive chronic pain care. Behavioral health treatment, such as cognitive-behavioral therapy (CBT) for pain, focuses on improved daily functioning and coping through several core treatment components such as psychoeducation about how the body processes pain, relaxation skills training, and cognitive strategies. Youth with chronic SCD pain need an evidence-based, culturally informed, adaptive treatment. Behavioral treatments that are tailored to patient and family needs are beneficial when patients may require different levels of care. Adaptive designs are more effective in improving health outcomes, satisfaction with treatment, and reducing healthcare use than standard protocols where patients receive a fixed one size fits all treatment that is not personalized to their needs; adaptive designs are also recommended for tailoring evidence-based interventions with culturally diverse populations. Adaptive treatments can integrate evidence-based strategies to address common co-morbid problems associated with chronic pain, such as elevated anxiety or depressive symptoms or sleep disturbance. Teaching parents problem-solving skills can reduce caregiver stress among families managing chronic pain and illness. This study utilizes an adaptive behavioral treatment to target psychosocial risk factors for youth with chronic SCD pain as a first step towards developing a stepped care model for SCD pain. The proposed treatment, called Back2Life, is innovative because it is culturally informed by families' and patients' feedback regarding their treatment preferences and targets psychological co-morbidities that are often excluded in clinical trials. The overarching hypothesis driving the proposed intervention is that disrupting the complex interacting psychosocial factors that can exacerbate chronic SCD pain will prevent and/or reduce poor health outcomes in children and adolescents with SCD. Reductions in inflammatory biomarkers are examined as an exploratory hypothesis and participants can consent to an optional blood draw for this portion of the study.
Interventions
BEHAVIORALBack2Life
The Back2Life intervention uses an adaptive treatment approach with module-based treatment sessions selected on the basis of baseline assessment (rather than a fixed treatment approach) to allow flexibility in tailoring treatment components to meet individual family needs. All youth participants will receive the standard 6-session pain coping skills training program, consisting of learning ways to cope with and manage chronic sickle cell pain. The standard program includes topics that were identified by young people with chronic sickle cell pain and their parents as important skills for all youth with chronic pain and sickle cell disease. In addition to the standard 6-session program, youth participants may receive an additional 1 to 4 sessions that may help with specific problems and/or co-morbidities related to pain. At least one parent or guardian is required to attend the sessions with their child.
Sponsors
National Heart, Lung, and Blood Institute (NHLBI)
Emory University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
SUPPORTIVE_CARE
Masking
NONE
Intervention model description
Youth and parent/caregiver dyads will participate in the same intervention.
Eligibility
Sex/Gender
ALL
Age
10 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
for Youth: * diagnosed with SCD (any genotype) * report chronic pain * speak and read English * have not initiated new disease modifying-treatments (e.g, hydroxyurea, Endari, voxelotor, crizanlizumab, chronic transfusions) or significantly increased dosages of any disease-modifying treatments in the past 3 months Inclusion Criteria for Parents or Caregivers: * speak and read English
Exclusion criteria
for Youth: * have comorbid medical conditions typically associated with pain but unrelated to SCD (e.g., rheumatologic disorders or inflammatory bowel disease) * are receiving chronic transfusion indicated for central nervous system risks and/or complications, previous overt strokes, or significant cognitive or developmental limitations, as per their healthcare provider or parent, that would impair completion of self-report measures or engagement in treatment sessions * received ≥ 3 sessions of outpatient psychological therapy for pain management in the 6 months prior to screening
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Dyads Completing the Study | Month 6 | Treatment feasibility was assessed by the number of participant dyads who complete the study. |
| Number of Dyads Completing Study Assignments Within Six Months | Month 6 | Treatment feasibility was assessed by the number of dyads completing the 6-Month study assignment. |
| Patient Reported Outcomes Measurement Information System (PROMIS) Pediatric Short Form Pain Interference Score | Baseline, Immediately Post-Treatment, Month 3, Month 6 | The PROMIS Pediatric Short Form for Pain Interference, Self- and Parent-Proxy Report, is an 8-item self-report measure assessing functional interference due to pain in the past 7 days. Total scores are standardized to a T-score with a mean of 50 and a standard deviation of 10, where higher scores indicate increased hindrance of life activities due to pain. |
| Sickle Cell Disease Pain Burden Interview-Youth (SCPBI-Y) Score | Baseline, Immediately Post-Treatment, Month 3, Month 6 | The Sickle Cell Pain Burden Interview for Youth, Self-Report is a 7-item, validated measure of pain burden in 7-21 year olds. Responses are given on a 5-point Likert scale where 0 = none and 4 = every. The patient self-reports the amount of days in the past month where pain occurred or pain impacted daily life. Total scores range from 0 to 28 and higher scores indicate a greater pain burden. |
| PROMIS Pediatric Short Form Pain Behaviors, Parent-Proxy Report Score | Baseline, Immediately Post-Treatment, Month 3, Month 6 | The PROMIS Pediatric Short Form Pain Behaviors, Parent-Proxy Report is an 8-item measure completed by parents that assesses pain behaviors displayed by their child in the past 7 days. Total scores are standardized to a T-score with a mean of 50 and a standard deviation of 10, where higher scores indicate increased behaviors due to pain. |
| Child Self-Efficacy Scale Score | Baseline, Immediately Post-Treatment, Month 3, Month 6 | Child Self-Efficacy Scale, Self- and Parent-Proxy Report is a well-established, 7-item measure of self-efficacy for functioning despite pain for 8-19 year olds. Respondents report how sure about their (or their child's) ability to perform certain daily tasks when they have pain, on a scale from 1 to 5 where 1 = very sure and 5 = very unsure. Total scores range from 7 to 35 and lower scores indicate greater self-efficacy. |
| Number of Participants With Positive Experiences During Participant Evaluation of the Intervention Interview | Immediately Post-Treatment | Treatment feasibility was assessed via a qualitative interview where participants were asked open ended questions. Participants were asked if they thought the Back2Life program helpful, if delivering the program through telemedicine was reasonable, and if the program was a reasonable approach for chronic pain management of sickle cell disease. |
| Treatment Evaluation Inventory-Short Form (TEI-SF) Score | Immediately Post-Treatment | The Treatment Evaluation Inventory-Short Form was completed at the end of treatment. It includes 9 items adapted to be specific to pediatric pain. Items are rated on a 5-point Likert scale ranging from 1 to 5. Total scores range from 9 to 45. Higher scores indicate increased acceptability with the study treatment. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Interleukin -1β (IL-1β) Concentration | Baseline, Month 3, Month 6 | Plasma concentration of the inflammatory biomarker IL-1β is assessed among youth participants who opted for blood sample collection. IL-1β increases in response to inflammation, pain, and autoimmune diseases. |
| IInterleukin 6 (IL-6) Concentration | Baseline, Month 3, Month 6 | Plasma concentration of the inflammatory biomarker IL-6 is assessed among youth participants who opted for blood sample collection. IL-6 is increased during injury or illness. |
| Interleukin 8 (IL-8) Concentration | Baseline, Month 3, Month 6 | Plasma concentration of the inflammatory biomarker IL-8 is assessed among youth participants who opted for blood sample collection. IL-8 is produced during the presence of inflammation. |
| Hospital Admissions Per Participant | 12 months prior to Baseline, 12 months post-treatment | Healthcare utilization was extracted from the medical record to document the mean number of hospital admissions for pain per participant during the 12 months preceding the intervention and the 12-month period post-treatment. |
| C-Reactive Protein (CRP) Concentration | Baseline, Month 3, Month 6 | Plasma concentration of the inflammatory biomarker CRP is assessed among youth participants who opted for blood sample collection. CRP increases in response to bodily inflammation. |
| Brain-Derived Neurotrophic Factor (BDNF) Concentration | Baseline, Month 3, Month 6 | Plasma concentration of the inflammatory biomarker BDNF is assessed among youth participants who opted for blood sample collection. BDNF expression is reduced when high bodily inflammation is present. |
| Interferon Gamma (IFN-y) Concentration | Baseline, Month 3, Month 6 | Plasma concentration of the inflammatory biomarker IFN-y is assessed among youth participants who opted for blood sample collection. IFN-y is involved with regulating immune and inflammatory responses. IFN-y concentration is elevated during illness. |
| Tumor Necrosis Factor - Alpha (TNF-α) Concentration | Baseline, Month 3, Month 6 | Plasma concentration of the inflammatory biomarker TNF-α is assessed among youth participants who opted for blood sample collection. TNF-α is a pro-inflammatory cytokine that regulates the inflammatory response and it is elevated during illness or injury. |
| Days Per Week of Opioid Use | Baseline, Immediately Post-Treatment, Month 3, Month 6 | Daily use of opioid pain medication was determined based on participant completion of daily diaries for 1-week at each assessment visit. Participants recorded opioid use daily (presence/absence). |
| Pediatric Inventory for Parents (PIP) Score | Baseline, Immediately Post-Treatment, Month 3, Month 6 | The Pediatric Inventory for Parents is a 42-item parent-reported measure of caregiver stress related to child chronic illness. Responses are given on a 5-point Likert scale where 1 = not at all and 5 = extremely. Total scores range from 42 to 210 and higher scores indicate greater caregiver stress. |
| Emergency Department Visits Per Participant | 12 months prior to Baseline, 12 months post-treatment | Healthcare utilization was extracted from the medical record to document the mean number of emergency department (ED) visits for pain per participant during the 12 months preceding the intervention period and the 12-month period post-treatment. |
| Adolescent Sleep Wake Scale (ASWS) Score | Baseline, Immediately Post-Treatment, Month 3, Month 6 | The Adolescent Sleep Wake Scale (ASWS) is a 28-item patient-reported describing the occurrence and frequency of various behavioral sleep characteristics over the past month. Responses are given on a 6-point Likert scale where 1 = always and 6 = never. Total scores are calculated as the mean score from each time and range from 1 to 6 where higher scores indicate better sleep quality. |
| PROMIS Pediatric Short Form Depressive Symptoms Score | Baseline, Immediately Post-Treatment, Month 3, Month 6 | The PROMIS Pediatric Short Form Depressive Symptoms questionnaire, Self- and Parent-Proxy Report is an 8-item measure designed for youth to assess self-reported symptoms of depression. Total scores are standardized to a T-score with a mean of 50 and a standard deviation of 10, where higher scores indicate increased depression. |
| Pain Catastrophizing Scale Score | Baseline, Immediately Post-Treatment, Month 3, Month 6 | The Pain Catastrophizing Scale, Child and Parent Report, is a 13-item well-validated self-report and parent-report measure of worried thoughts about pain. Items are answered on a 5-point scale where 0 = not true at all and 4 = very true. Total scores range from 0 to 52 and higher scores indicate increased catastrophic thinking. |
| Pain Stages of Change Questionnaire (PSOCQ) Action Scale Score | Baseline, Immediately Post-Treatment, Month 3, Month 6 | The Pain Stages of Change Questionnaire, Adolescent and Parent Report is a 30-item measure designed to evaluate parent and adolescent perceptions of readiness to adopt a self-management approach to pain. Responses are given on a 5-point scale where 1 = strongly disagree and 5 = strongly agree. Average scores are obtained for categories of Pre-contemplation (believing pain management is solely the responsibility of medical professionals), Contemplation (considering using self-management for pain), Action (starts taking ownership of pain control and self-management of pain), and Maintenance (continuous development and self-management of chronic pain); the category with the highest score indicates where the youth participant is in terms of stages of change related to pain management. For this outcome measure, the average scores for the Action scale are reported. Total scores for the Action scale range from 1 to 5 and higher scores indicate increased readiness to self-manage pain. |
Countries
United States
Participant flow
Recruitment details
Participants were recruited from Children's Healthcare of Atlanta, Emory Children's Center, and Children's Healthcare of Atlanta at Hughes Spalding in Atlanta, Georgia, USA. Participant enrollment began January 27, 2021. Data collection for the 6 Month time point ended August 23, 2023 and data collection for the 12 Month time point was complete on May 18, 2024.
Participants by arm
| Arm | Count |
|---|---|
| Back2Life Program for Youth Youth with chronic SCD pain receiving an adaptive cognitive behavioral treatment program for pain coping skills. | 20 |
| Back2Life for Parents or Caregivers Parents or caregivers of youth with chronic SCD pain receiving an adaptive cognitive behavioral treatment program for pain coping skills. | 20 |
| Total | 40 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Lost to Follow-up | 4 | 4 |
Baseline characteristics
| Characteristic | Back2Life Program for Youth | Total | Back2Life for Parents or Caregivers |
|---|---|---|---|
| Age, Continuous | 15.8 years STANDARD_DEVIATION 1.7 | 26.9 years STANDARD_DEVIATION 13.4 | 41.6 years STANDARD_DEVIATION 5.1 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 0 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 18 Participants | 33 Participants | 15 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 2 Participants | 7 Participants | 5 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 20 Participants | 35 Participants | 15 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 5 Participants | 5 Participants |
| Race (NIH/OMB) White | 0 Participants | 0 Participants | 0 Participants |
| Region of Enrollment United States | 20 Participants | 40 Participants | 20 Participants |
| Sex: Female, Male Female | 9 Participants | 28 Participants | 19 Participants |
| Sex: Female, Male Male | 11 Participants | 12 Participants | 1 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 20 | 0 / 20 |
| other Total, other adverse events | 0 / 20 | 0 / 20 |
| serious Total, serious adverse events | 0 / 20 | 0 / 20 |
Outcome results
Primary
Child Self-Efficacy Scale Score
Child Self-Efficacy Scale, Self- and Parent-Proxy Report is a well-established, 7-item measure of self-efficacy for functioning despite pain for 8-19 year olds. Respondents report how sure about their (or their child's) ability to perform certain daily tasks when they have pain, on a scale from 1 to 5 where 1 = very sure and 5 = very unsure. Total scores range from 7 to 35 and lower scores indicate greater self-efficacy.
Time frame: Baseline, Immediately Post-Treatment, Month 3, Month 6
Population: The analysis population includes participants remaining in the study at the indicated time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Back2Life Program for Youth | Child Self-Efficacy Scale Score | Baseline | 23.0 score on a scale | Standard Deviation 8.5 |
| Back2Life Program for Youth | Child Self-Efficacy Scale Score | Immediately Post-Treatment | 20.0 score on a scale | Standard Deviation 5.8 |
| Back2Life Program for Youth | Child Self-Efficacy Scale Score | Month 3 | 18.3 score on a scale | Standard Deviation 2.6 |
| Back2Life Program for Youth | Child Self-Efficacy Scale Score | Month 6 | 17.0 score on a scale | Standard Deviation 4 |
| Back2Life for Parents or Caregivers | Child Self-Efficacy Scale Score | Month 6 | 21.6 score on a scale | Standard Deviation 8.9 |
| Back2Life for Parents or Caregivers | Child Self-Efficacy Scale Score | Baseline | 24.6 score on a scale | Standard Deviation 8.2 |
| Back2Life for Parents or Caregivers | Child Self-Efficacy Scale Score | Month 3 | 24.3 score on a scale | Standard Deviation 5.5 |
| Back2Life for Parents or Caregivers | Child Self-Efficacy Scale Score | Immediately Post-Treatment | 24.1 score on a scale | Standard Deviation 8.1 |
Primary
Number of Dyads Completing Study Assignments Within Six Months
Treatment feasibility was assessed by the number of dyads completing the 6-Month study assignment.
Time frame: Month 6
Population: The analysis population includes youth and caregivers dyad participants who completed the Back2Life program.
| Arm | Measure | Value (COUNT_OF_UNITS) |
|---|---|---|
| Back2Life Program for Youth | Number of Dyads Completing Study Assignments Within Six Months | 16 dyads |
Primary
Number of Dyads Completing the Study
Treatment feasibility was assessed by the number of participant dyads who complete the study.
Time frame: Month 6
| Arm | Measure | Value (COUNT_OF_UNITS) |
|---|---|---|
| Back2Life Program for Youth | Number of Dyads Completing the Study | 16 dyads |
Primary
Number of Participants With Positive Experiences During Participant Evaluation of the Intervention Interview
Treatment feasibility was assessed via a qualitative interview where participants were asked open ended questions. Participants were asked if they thought the Back2Life program helpful, if delivering the program through telemedicine was reasonable, and if the program was a reasonable approach for chronic pain management of sickle cell disease.
Time frame: Immediately Post-Treatment
Population: The analysis population includes participants who completed the Back2Life program.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Back2Life Program for Youth | Number of Participants With Positive Experiences During Participant Evaluation of the Intervention Interview | Reported program was helpful | 15 Participants |
| Back2Life Program for Youth | Number of Participants With Positive Experiences During Participant Evaluation of the Intervention Interview | Reported a positive experience with telehealth delivery | 16 Participants |
| Back2Life Program for Youth | Number of Participants With Positive Experiences During Participant Evaluation of the Intervention Interview | Reported the program was relevant to sickle cell disease | 16 Participants |
| Back2Life for Parents or Caregivers | Number of Participants With Positive Experiences During Participant Evaluation of the Intervention Interview | Reported program was helpful | 16 Participants |
| Back2Life for Parents or Caregivers | Number of Participants With Positive Experiences During Participant Evaluation of the Intervention Interview | Reported a positive experience with telehealth delivery | 16 Participants |
| Back2Life for Parents or Caregivers | Number of Participants With Positive Experiences During Participant Evaluation of the Intervention Interview | Reported the program was relevant to sickle cell disease | 16 Participants |
Primary
Patient Reported Outcomes Measurement Information System (PROMIS) Pediatric Short Form Pain Interference Score
The PROMIS Pediatric Short Form for Pain Interference, Self- and Parent-Proxy Report, is an 8-item self-report measure assessing functional interference due to pain in the past 7 days. Total scores are standardized to a T-score with a mean of 50 and a standard deviation of 10, where higher scores indicate increased hindrance of life activities due to pain.
Time frame: Baseline, Immediately Post-Treatment, Month 3, Month 6
Population: The analysis population includes participants remaining in the study at the indicated time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Back2Life Program for Youth | Patient Reported Outcomes Measurement Information System (PROMIS) Pediatric Short Form Pain Interference Score | Baseline | 61.5 T-score | Standard Deviation 10.8 |
| Back2Life Program for Youth | Patient Reported Outcomes Measurement Information System (PROMIS) Pediatric Short Form Pain Interference Score | Immediately Post-Treatment | 58.2 T-score | Standard Deviation 12.5 |
| Back2Life Program for Youth | Patient Reported Outcomes Measurement Information System (PROMIS) Pediatric Short Form Pain Interference Score | Month 3 | 50.4 T-score | Standard Deviation 11.6 |
| Back2Life Program for Youth | Patient Reported Outcomes Measurement Information System (PROMIS) Pediatric Short Form Pain Interference Score | Month 6 | 50.3 T-score | Standard Deviation 14.1 |
| Back2Life for Parents or Caregivers | Patient Reported Outcomes Measurement Information System (PROMIS) Pediatric Short Form Pain Interference Score | Month 6 | 57.7 T-score | Standard Deviation 11.9 |
| Back2Life for Parents or Caregivers | Patient Reported Outcomes Measurement Information System (PROMIS) Pediatric Short Form Pain Interference Score | Baseline | 62.1 T-score | Standard Deviation 11.1 |
| Back2Life for Parents or Caregivers | Patient Reported Outcomes Measurement Information System (PROMIS) Pediatric Short Form Pain Interference Score | Month 3 | 53.7 T-score | Standard Deviation 12.9 |
| Back2Life for Parents or Caregivers | Patient Reported Outcomes Measurement Information System (PROMIS) Pediatric Short Form Pain Interference Score | Immediately Post-Treatment | 59.4 T-score | Standard Deviation 12.2 |
Primary
PROMIS Pediatric Short Form Pain Behaviors, Parent-Proxy Report Score
The PROMIS Pediatric Short Form Pain Behaviors, Parent-Proxy Report is an 8-item measure completed by parents that assesses pain behaviors displayed by their child in the past 7 days. Total scores are standardized to a T-score with a mean of 50 and a standard deviation of 10, where higher scores indicate increased behaviors due to pain.
Time frame: Baseline, Immediately Post-Treatment, Month 3, Month 6
Population: The analysis population includes parent or caregiver participants remaining in the study at the indicated time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Back2Life Program for Youth | PROMIS Pediatric Short Form Pain Behaviors, Parent-Proxy Report Score | Baseline | 52.7 T-score | Standard Deviation 20.6 |
| Back2Life Program for Youth | PROMIS Pediatric Short Form Pain Behaviors, Parent-Proxy Report Score | Immediately Post-Treatment | 52.0 T-score | Standard Deviation 20.6 |
| Back2Life Program for Youth | PROMIS Pediatric Short Form Pain Behaviors, Parent-Proxy Report Score | Month 3 | 46.2 T-score | Standard Deviation 22 |
| Back2Life Program for Youth | PROMIS Pediatric Short Form Pain Behaviors, Parent-Proxy Report Score | Month 6 | 50.8 T-score | Standard Deviation 25.9 |
Primary
Sickle Cell Disease Pain Burden Interview-Youth (SCPBI-Y) Score
The Sickle Cell Pain Burden Interview for Youth, Self-Report is a 7-item, validated measure of pain burden in 7-21 year olds. Responses are given on a 5-point Likert scale where 0 = none and 4 = every. The patient self-reports the amount of days in the past month where pain occurred or pain impacted daily life. Total scores range from 0 to 28 and higher scores indicate a greater pain burden.
Time frame: Baseline, Immediately Post-Treatment, Month 3, Month 6
Population: The analysis population includes youth participants remaining in the study at the indicated time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Back2Life Program for Youth | Sickle Cell Disease Pain Burden Interview-Youth (SCPBI-Y) Score | Baseline | 13.8 score on a scale | Standard Deviation 7.5 |
| Back2Life Program for Youth | Sickle Cell Disease Pain Burden Interview-Youth (SCPBI-Y) Score | Immediately Post-Treatment | 9.5 score on a scale | Standard Deviation 7.7 |
| Back2Life Program for Youth | Sickle Cell Disease Pain Burden Interview-Youth (SCPBI-Y) Score | Month 3 | 9.1 score on a scale | Standard Deviation 5.3 |
| Back2Life Program for Youth | Sickle Cell Disease Pain Burden Interview-Youth (SCPBI-Y) Score | Month 6 | 7.0 score on a scale | Standard Deviation 7 |
Primary
Treatment Evaluation Inventory-Short Form (TEI-SF) Score
The Treatment Evaluation Inventory-Short Form was completed at the end of treatment. It includes 9 items adapted to be specific to pediatric pain. Items are rated on a 5-point Likert scale ranging from 1 to 5. Total scores range from 9 to 45. Higher scores indicate increased acceptability with the study treatment.
Time frame: Immediately Post-Treatment
Population: The analysis population includes participants who completed the Back2Life program.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Back2Life Program for Youth | Treatment Evaluation Inventory-Short Form (TEI-SF) Score | 32.0 score on a scale | Standard Deviation 6.1 |
| Back2Life for Parents or Caregivers | Treatment Evaluation Inventory-Short Form (TEI-SF) Score | 31.5 score on a scale | Standard Deviation 4.3 |
Secondary
Adolescent Sleep Wake Scale (ASWS) Score
The Adolescent Sleep Wake Scale (ASWS) is a 28-item patient-reported describing the occurrence and frequency of various behavioral sleep characteristics over the past month. Responses are given on a 6-point Likert scale where 1 = always and 6 = never. Total scores are calculated as the mean score from each time and range from 1 to 6 where higher scores indicate better sleep quality.
Time frame: Baseline, Immediately Post-Treatment, Month 3, Month 6
Population: The analysis population includes participants remaining in the study at the indicated time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Back2Life Program for Youth | Adolescent Sleep Wake Scale (ASWS) Score | Baseline | 2.9 score on a scale | Standard Deviation 0.4 |
| Back2Life Program for Youth | Adolescent Sleep Wake Scale (ASWS) Score | Immediately Post-Treatment | 3.1 score on a scale | Standard Deviation 0.5 |
| Back2Life Program for Youth | Adolescent Sleep Wake Scale (ASWS) Score | Month 3 | 2.8 score on a scale | Standard Deviation 0.2 |
| Back2Life Program for Youth | Adolescent Sleep Wake Scale (ASWS) Score | Month 6 | 3.0 score on a scale | Standard Deviation 0.3 |
Secondary
Brain-Derived Neurotrophic Factor (BDNF) Concentration
Plasma concentration of the inflammatory biomarker BDNF is assessed among youth participants who opted for blood sample collection. BDNF expression is reduced when high bodily inflammation is present.
Time frame: Baseline, Month 3, Month 6
Population: This analysis includes youth participants who provided an analyzable blood sample for the indicated time point. Collection of biomarkers was an optional study activity for participants, requiring separate consent. A total of 4 participants contributed at least one sample during the course of the study. Not all collected samples could be analyzed due to laboratory issues, such as insufficient sample volume.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Back2Life Program for Youth | Brain-Derived Neurotrophic Factor (BDNF) Concentration | Baseline | 4052.33 pg/mL | Standard Deviation 1445.3 |
| Back2Life Program for Youth | Brain-Derived Neurotrophic Factor (BDNF) Concentration | Month 3 | 3823.00 pg/mL | Standard Deviation 0 |
| Back2Life Program for Youth | Brain-Derived Neurotrophic Factor (BDNF) Concentration | Month 6 | 4867.50 pg/mL | Standard Deviation 1853.33 |
Secondary
C-Reactive Protein (CRP) Concentration
Plasma concentration of the inflammatory biomarker CRP is assessed among youth participants who opted for blood sample collection. CRP increases in response to bodily inflammation.
Time frame: Baseline, Month 3, Month 6
Population: This analysis includes youth participants who provided an analyzable blood sample for the indicated time point. Collection of biomarkers was an optional study activity for participants, requiring separate consent. A total of 4 participants contributed at least one sample during the course of the study. Not all collected samples could be analyzed due to laboratory issues, such as insufficient sample volume.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Back2Life Program for Youth | C-Reactive Protein (CRP) Concentration | Baseline | 1710.74 nanograms per milliliter (ng/mL) | Standard Deviation 746.33 |
| Back2Life Program for Youth | C-Reactive Protein (CRP) Concentration | Month 3 | 4795.63 nanograms per milliliter (ng/mL) | Standard Deviation 0 |
| Back2Life Program for Youth | C-Reactive Protein (CRP) Concentration | Month 6 | 5206.06 nanograms per milliliter (ng/mL) | Standard Deviation 3271.69 |
Secondary
Days Per Week of Opioid Use
Daily use of opioid pain medication was determined based on participant completion of daily diaries for 1-week at each assessment visit. Participants recorded opioid use daily (presence/absence).
Time frame: Baseline, Immediately Post-Treatment, Month 3, Month 6
Population: The analysis population includes youth participants remaining in the study at the indicated time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Back2Life Program for Youth | Days Per Week of Opioid Use | Baseline | 3.9 days/week | Standard Deviation 2.1 |
| Back2Life Program for Youth | Days Per Week of Opioid Use | Immediately Post-Treatment | 1.5 days/week | Standard Deviation 1.4 |
| Back2Life Program for Youth | Days Per Week of Opioid Use | Month 3 | 1.8 days/week | Standard Deviation 0.75 |
| Back2Life Program for Youth | Days Per Week of Opioid Use | Month 6 | 1.2 days/week | Standard Deviation 0.75 |
Secondary
Emergency Department Visits Per Participant
Healthcare utilization was extracted from the medical record to document the mean number of emergency department (ED) visits for pain per participant during the 12 months preceding the intervention period and the 12-month period post-treatment.
Time frame: 12 months prior to Baseline, 12 months post-treatment
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Back2Life Program for Youth | Emergency Department Visits Per Participant | 12 Months Pre-Treatment | 0.86 visits per participant | Standard Deviation 1.06 |
| Back2Life Program for Youth | Emergency Department Visits Per Participant | 12 Months Post-Treatment | 0.9 visits per participant | Standard Deviation 1.07 |
Secondary
Hospital Admissions Per Participant
Healthcare utilization was extracted from the medical record to document the mean number of hospital admissions for pain per participant during the 12 months preceding the intervention and the 12-month period post-treatment.
Time frame: 12 months prior to Baseline, 12 months post-treatment
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Back2Life Program for Youth | Hospital Admissions Per Participant | 12 Months Pre-Treatment | 1.19 admissions per participant | Standard Deviation 1.29 |
| Back2Life Program for Youth | Hospital Admissions Per Participant | 12 Months Post-Treatment | 1.25 admissions per participant | Standard Deviation 1.29 |
Secondary
IInterleukin 6 (IL-6) Concentration
Plasma concentration of the inflammatory biomarker IL-6 is assessed among youth participants who opted for blood sample collection. IL-6 is increased during injury or illness.
Time frame: Baseline, Month 3, Month 6
Population: This analysis includes youth participants who provided an analyzable blood sample for the indicated time point. Collection of biomarkers was an optional study activity for participants, requiring separate consent. A total of 4 participants contributed at least one sample during the course of the study. Not all collected samples could be analyzed due to laboratory issues, such as insufficient sample volume.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Back2Life Program for Youth | IInterleukin 6 (IL-6) Concentration | Month 6 | 1.91 pg/mL | Standard Deviation 1.8 |
| Back2Life Program for Youth | IInterleukin 6 (IL-6) Concentration | Baseline | 1.88 pg/mL | Standard Deviation 2.31 |
| Back2Life Program for Youth | IInterleukin 6 (IL-6) Concentration | Month 3 | 2.24 pg/mL | Standard Deviation 0 |
Secondary
Interferon Gamma (IFN-y) Concentration
Plasma concentration of the inflammatory biomarker IFN-y is assessed among youth participants who opted for blood sample collection. IFN-y is involved with regulating immune and inflammatory responses. IFN-y concentration is elevated during illness.
Time frame: Baseline, Month 3, Month 6
Population: This analysis includes youth participants who provided an analyzable blood sample for the indicated time point. Collection of biomarkers was an optional study activity for participants, requiring separate consent. A total of 4 participants contributed at least one sample during the course of the study. Not all collected samples could be analyzed due to laboratory issues, such as insufficient sample volume.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Back2Life Program for Youth | Interferon Gamma (IFN-y) Concentration | Baseline | 19.26 pg/mL | Standard Deviation 17.99 |
| Back2Life Program for Youth | Interferon Gamma (IFN-y) Concentration | Month 3 | 20 pg/mL | Standard Deviation 0 |
| Back2Life Program for Youth | Interferon Gamma (IFN-y) Concentration | Month 6 | 19.70 pg/mL | Standard Deviation 10.04 |
Secondary
Interleukin -1β (IL-1β) Concentration
Plasma concentration of the inflammatory biomarker IL-1β is assessed among youth participants who opted for blood sample collection. IL-1β increases in response to inflammation, pain, and autoimmune diseases.
Time frame: Baseline, Month 3, Month 6
Population: This analysis includes youth participants who provided an analyzable blood sample for the indicated time point. Collection of biomarkers was an optional study activity for participants, requiring separate consent. A total of 4 participants contributed at least one sample during the course of the study. Not all collected samples could be analyzed due to laboratory issues, such as insufficient sample volume.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Back2Life Program for Youth | Interleukin -1β (IL-1β) Concentration | Baseline | 1.25 picograms per milliliter (pg/mL) | Standard Deviation 1.21 |
| Back2Life Program for Youth | Interleukin -1β (IL-1β) Concentration | Month 3 | 1.15 picograms per milliliter (pg/mL) | Standard Deviation 0 |
| Back2Life Program for Youth | Interleukin -1β (IL-1β) Concentration | Month 6 | 0.95 picograms per milliliter (pg/mL) | Standard Deviation 0.88 |
Secondary
Interleukin 8 (IL-8) Concentration
Plasma concentration of the inflammatory biomarker IL-8 is assessed among youth participants who opted for blood sample collection. IL-8 is produced during the presence of inflammation.
Time frame: Baseline, Month 3, Month 6
Population: This analysis includes youth participants who provided an analyzable blood sample for the indicated time point. Collection of biomarkers was an optional study activity for participants, requiring separate consent. A total of 4 participants contributed at least one sample during the course of the study. Not all collected samples could be analyzed due to laboratory issues, such as insufficient sample volume.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Back2Life Program for Youth | Interleukin 8 (IL-8) Concentration | Baseline | 10.28 pg/mL | Standard Deviation 8.26 |
| Back2Life Program for Youth | Interleukin 8 (IL-8) Concentration | Month 3 | 27.70 pg/mL | Standard Deviation 0 |
| Back2Life Program for Youth | Interleukin 8 (IL-8) Concentration | Month 6 | 14.28 pg/mL | Standard Deviation 14.32 |
Secondary
Pain Catastrophizing Scale Score
The Pain Catastrophizing Scale, Child and Parent Report, is a 13-item well-validated self-report and parent-report measure of worried thoughts about pain. Items are answered on a 5-point scale where 0 = not true at all and 4 = very true. Total scores range from 0 to 52 and higher scores indicate increased catastrophic thinking.
Time frame: Baseline, Immediately Post-Treatment, Month 3, Month 6
Population: The analysis population includes participants remaining in the study at the indicated time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Back2Life Program for Youth | Pain Catastrophizing Scale Score | Baseline | 27.8 score on a scale | Standard Deviation 12 |
| Back2Life Program for Youth | Pain Catastrophizing Scale Score | Immediately Post-Treatment | 24.9 score on a scale | Standard Deviation 10.7 |
| Back2Life Program for Youth | Pain Catastrophizing Scale Score | Month 3 | 18.1 score on a scale | Standard Deviation 7.2 |
| Back2Life Program for Youth | Pain Catastrophizing Scale Score | Month 6 | 15.3 score on a scale | Standard Deviation 4.9 |
| Back2Life for Parents or Caregivers | Pain Catastrophizing Scale Score | Month 6 | 34.3 score on a scale | Standard Deviation 7.9 |
| Back2Life for Parents or Caregivers | Pain Catastrophizing Scale Score | Baseline | 33.3 score on a scale | Standard Deviation 12.4 |
| Back2Life for Parents or Caregivers | Pain Catastrophizing Scale Score | Month 3 | 25.6 score on a scale | Standard Deviation 13.7 |
| Back2Life for Parents or Caregivers | Pain Catastrophizing Scale Score | Immediately Post-Treatment | 35.8 score on a scale | Standard Deviation 15.3 |
Secondary
Pain Stages of Change Questionnaire (PSOCQ) Action Scale Score
The Pain Stages of Change Questionnaire, Adolescent and Parent Report is a 30-item measure designed to evaluate parent and adolescent perceptions of readiness to adopt a self-management approach to pain. Responses are given on a 5-point scale where 1 = strongly disagree and 5 = strongly agree. Average scores are obtained for categories of Pre-contemplation (believing pain management is solely the responsibility of medical professionals), Contemplation (considering using self-management for pain), Action (starts taking ownership of pain control and self-management of pain), and Maintenance (continuous development and self-management of chronic pain); the category with the highest score indicates where the youth participant is in terms of stages of change related to pain management. For this outcome measure, the average scores for the Action scale are reported. Total scores for the Action scale range from 1 to 5 and higher scores indicate increased readiness to self-manage pain.
Time frame: Baseline, Immediately Post-Treatment, Month 3, Month 6
Population: The analysis population includes participants remaining in the study at the indicated time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Back2Life Program for Youth | Pain Stages of Change Questionnaire (PSOCQ) Action Scale Score | Baseline | 3.7 score on a scale | Standard Deviation 0.58 |
| Back2Life Program for Youth | Pain Stages of Change Questionnaire (PSOCQ) Action Scale Score | Immediately Post-Treatment | 3.9 score on a scale | Standard Deviation 0.69 |
| Back2Life Program for Youth | Pain Stages of Change Questionnaire (PSOCQ) Action Scale Score | Month 3 | 4.0 score on a scale | Standard Deviation 0.45 |
| Back2Life Program for Youth | Pain Stages of Change Questionnaire (PSOCQ) Action Scale Score | Month 6 | 4.2 score on a scale | Standard Deviation 0.64 |
| Back2Life for Parents or Caregivers | Pain Stages of Change Questionnaire (PSOCQ) Action Scale Score | Month 6 | 3.8 score on a scale | Standard Deviation 0.98 |
| Back2Life for Parents or Caregivers | Pain Stages of Change Questionnaire (PSOCQ) Action Scale Score | Baseline | 3.9 score on a scale | Standard Deviation 0.45 |
| Back2Life for Parents or Caregivers | Pain Stages of Change Questionnaire (PSOCQ) Action Scale Score | Month 3 | 3.9 score on a scale | Standard Deviation 0.42 |
| Back2Life for Parents or Caregivers | Pain Stages of Change Questionnaire (PSOCQ) Action Scale Score | Immediately Post-Treatment | 4.3 score on a scale | Standard Deviation 0.44 |
Secondary
Pediatric Inventory for Parents (PIP) Score
The Pediatric Inventory for Parents is a 42-item parent-reported measure of caregiver stress related to child chronic illness. Responses are given on a 5-point Likert scale where 1 = not at all and 5 = extremely. Total scores range from 42 to 210 and higher scores indicate greater caregiver stress.
Time frame: Baseline, Immediately Post-Treatment, Month 3, Month 6
Population: The analysis population includes participants remaining in the study at the indicated time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Back2Life Program for Youth | Pediatric Inventory for Parents (PIP) Score | Baseline | 104.5 score on a scale | Standard Deviation 29.3 |
| Back2Life Program for Youth | Pediatric Inventory for Parents (PIP) Score | Immediately Post-Treatment | 91.0 score on a scale | Standard Deviation 46.7 |
| Back2Life Program for Youth | Pediatric Inventory for Parents (PIP) Score | Month 3 | 72.0 score on a scale | Standard Deviation 39.8 |
| Back2Life Program for Youth | Pediatric Inventory for Parents (PIP) Score | Month 6 | 71.0 score on a scale | Standard Deviation 32 |
Secondary
PROMIS Pediatric Short Form Depressive Symptoms Score
The PROMIS Pediatric Short Form Depressive Symptoms questionnaire, Self- and Parent-Proxy Report is an 8-item measure designed for youth to assess self-reported symptoms of depression. Total scores are standardized to a T-score with a mean of 50 and a standard deviation of 10, where higher scores indicate increased depression.
Time frame: Baseline, Immediately Post-Treatment, Month 3, Month 6
Population: The analysis population includes participants remaining in the study at the indicated time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Back2Life Program for Youth | PROMIS Pediatric Short Form Depressive Symptoms Score | Baseline | 71.1 T-score | Standard Deviation 6.3 |
| Back2Life Program for Youth | PROMIS Pediatric Short Form Depressive Symptoms Score | Immediately Post-Treatment | 68.9 T-score | Standard Deviation 7 |
| Back2Life Program for Youth | PROMIS Pediatric Short Form Depressive Symptoms Score | Month 3 | 65.5 T-score | Standard Deviation 4.9 |
| Back2Life Program for Youth | PROMIS Pediatric Short Form Depressive Symptoms Score | Month 6 | 65.1 T-score | Standard Deviation 4.5 |
| Back2Life for Parents or Caregivers | PROMIS Pediatric Short Form Depressive Symptoms Score | Month 6 | 47.9 T-score | Standard Deviation 12.3 |
| Back2Life for Parents or Caregivers | PROMIS Pediatric Short Form Depressive Symptoms Score | Baseline | 55.4 T-score | Standard Deviation 10.9 |
| Back2Life for Parents or Caregivers | PROMIS Pediatric Short Form Depressive Symptoms Score | Month 3 | 48.2 T-score | Standard Deviation 12 |
| Back2Life for Parents or Caregivers | PROMIS Pediatric Short Form Depressive Symptoms Score | Immediately Post-Treatment | 52.6 T-score | Standard Deviation 16.2 |
Secondary
Tumor Necrosis Factor - Alpha (TNF-α) Concentration
Plasma concentration of the inflammatory biomarker TNF-α is assessed among youth participants who opted for blood sample collection. TNF-α is a pro-inflammatory cytokine that regulates the inflammatory response and it is elevated during illness or injury.
Time frame: Baseline, Month 3, Month 6
Population: This analysis includes youth participants who provided an analyzable blood sample for the indicated time point. Collection of biomarkers was an optional study activity for participants, requiring separate consent. A total of 4 participants contributed at least one sample during the course of the study. Not all collected samples could be analyzed due to laboratory issues, such as insufficient sample volume.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Back2Life Program for Youth | Tumor Necrosis Factor - Alpha (TNF-α) Concentration | Baseline | 2.05 pg/mL | Standard Deviation 2.38 |
| Back2Life Program for Youth | Tumor Necrosis Factor - Alpha (TNF-α) Concentration | Month 3 | 2.57 pg/mL | Standard Deviation 0 |
| Back2Life Program for Youth | Tumor Necrosis Factor - Alpha (TNF-α) Concentration | Month 6 | 2.04 pg/mL | Standard Deviation 1.93 |