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| ID | Type | Description | Link |
|---|---|---|---|
| 1R01NR017206-01 | U.S. NIH Grant/Contract | View source |
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| Name | Class |
|---|---|
| National Institute of Nursing Research (NINR) | NIH |
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Many youth with chronic disease have difficulty taking medication every day and therefore do not receive full benefit from treatment. Sickle Cell Disease (SCD) is an inherited blood disease that affects African Americans and other underserved communities. Hydroxyurea (HU) is the sole FDA-approved drug therapy for SCD and is highly effective and improves quality of life. The proposed study, a 5-site four-year randomized control trial (RCT), builds upon the investigators' recent feasibility study of the same title. Overall goals are reducing barriers to HU use and improving adherence for youth 10-18 years through creation of a daily medication habit. The goal of the proposed multi-site study is to test the efficacy of the HABIT intervention at 6 months and sustainability of the effect at 12 months.
Barriers to medication adherence are common in youth with chronic illness and are a source of racial/ethnic disparities in underserved communities. An inherited blood disease, Sickle Cell Disease (SCD) is characterized by chronic and acute illness and reduced quality of life (QOL). It affects African Americans and other underserved communities. Hydroxyurea (HU) is the sole FDA-approved drug therapy for SCD and is highly effective and improves QOL. Poor adherence is common among youth and young adults with SCD.
The importance of poor medication adherence, use of community-based health workers (CHWs) to bridge the gap between health services and underserved parent-youth dyads affected by SCD, the strength of the science, the success of the investigators' multi-ethnic feasibility study, and the potential application of study findings to youth with other serious chronic illnesses speak to the importance of this trial.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Control Group | Active Comparator | Dyads randomized to the control group will receive: Standard care and Education handouts. |
|
| Intervention Group | Experimental | In addition to standard care and education handouts, dyads randomized to the intervention group will receive the HABIT intervention, which includes CHW support and tailored text messages. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| HABIT Intervention | Behavioral | Dyads randomized to the intervention group will receive the HABIT intervention, which includes CHW support and tailored text messages. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Mean Change in Biomarker Fetal Hemoglobin (HbF) | A serum biomarker obtained from youth used to measure adherence to hydroxyurea | Baseline, 6 months, 12 months |
| Mean Change in Proportion of Days Covered (PDC) by Hydroxyurea | The days covered by hydroxyurea was assessed using youth prescription refill data and was used to measure hydroxyurea adherence. The baseline measure is the proportion of days covered by hydroxyurea in the year prior to study enrollment, using prescription refill data. | Baseline, 6 months, 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| Mean Change in Youth Score on Peds Quality of Life (Generic Quality of Life) | Used to measure health-related quality of life. Scores range from 0-100 with a higher score indicating a better quality of life. | Baseline, 4 months, 9 months and 12 months |
| Mean Change in Youth Score on PedsQL Sickle Cell Disease Module (Disease Specific Quality of Life) |
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Inclusion Criteria - Youth:
Inclusion Criteria - Parent:
Exclusion Criteria - Youth:
Exclusion Criteria - Parent:
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| Name | Affiliation | Role |
|---|---|---|
| Arlene Smaldone, PhD, CPNP-PC | Columbia University School of Nursing | Principal Investigator |
| Nancy S Green, MD | Columbia University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Feinstein Institute for Medical Research | Manhasset | New York | 11030 | United States | ||
| Columbia University Irving Medical Center |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38015138 | Background | Bruzzese JM, Usseglio J, Iannacci-Manasia L, Diggs KA, Smaldone AM, Green NS. Mental and Emotional Health of Caregivers of Youth with Sickle Cell Disease: A Systematic Review. J Health Care Poor Underserved. 2023;34(3):1070-1104. | |
| 35856776 | Background | Askew MA, Smaldone AM, Gold MA, Smith-Whitley K, Strouse JJ, Jin Z, Green NS. Pediatric hematology providers' contraceptive practices for female adolescents and young adults with sickle cell disease: A national survey. Pediatr Blood Cancer. 2022 Oct;69(10):e29877. doi: 10.1002/pbc.29877. Epub 2022 Jul 20. |
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24 Dyads were randomized to the intervention (24 parents + 24 youth) 26 Dyads were randomized to the control (26 parents + 26 youth)
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| ID | Title | Description |
|---|---|---|
| FG000 | Intervention Group | In addition to standard care and education handouts, dyads randomized to the intervention group will receive the HABIT intervention, which includes community health workers (CHW) support and tailored text messages. |
| FG001 | Control Group | Dyads randomized to the control group will receive: Standard care and Education handouts. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
The following variables were collected for children: Age, Sex, Race, Ethnicity, No other chronic conditions besides sickle cell disease, Number of emergency room visits, Number of hospitalizations, Biomarker fetal hemoglobin, Proportion of days covered by hydroxyurea The following variables were collected for parent/primary caregiver: Parent status, Parent education, Caregiver affected by sickle cell disease, Other people at home affected by sickle cell disease
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| ID | Title | Description |
|---|---|---|
| BG000 | Intervention Group | In addition to standard care and education handouts, dyads randomized to the intervention group will receive the HABIT intervention, which includes CHW support and tailored text messages. |
| BG001 | Control Group |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Mean Change in Biomarker Fetal Hemoglobin (HbF) | A serum biomarker obtained from youth used to measure adherence to hydroxyurea | Change between 6-12 months: 22 of 24 intervention group participants analyzed as blood samples were collected at both timepoints for 22 of 24 participants, due to difficulties coming into clinic during the COVID-19 pandemic. | Posted | Mean | Standard Deviation | Percent fetal hemoglobin | Baseline, 6 months, 12 months |
|
12 months
AE data were only collected on youth
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Control Group | Dyads randomized to the control group will receive: Standard care and Education handouts. |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Arlene Smaldone, PhD, RN | Columbia University School of Nursing | 212-342-3048 | ams130@columbia.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Jul 14, 2020 | Mar 6, 2024 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| D055118 | Medication Adherence |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
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| ID | Term |
|---|---|
| D059039 | Standard of Care |
| ID | Term |
|---|---|
| D019984 | Quality Indicators, Health Care |
| D011787 | Quality of Health Care |
| D006298 | Health Services Administration |
| D017530 | Health Care Quality, Access, and Evaluation |
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| Standard of Care | Other | Standard of care used to treat patients with SCD |
|
| Education materials | Other | Education materials provided to all patients enrolled in the trial |
|
Used to measure sickle cell disease specific health-related quality of life. Scores range from 0-100 with a higher score indicating a better quality of life. |
| Baseline, 9 months and 12 months |
| Mean Change in Parent Youth Concordance Regarding Self-management Responsibility | Concordance between parent and youth scores Sickle Cell Family Responsibility scores for 11 items measuring self-management tasks. Scores range from 0-11 with a higher score indicating better concordance. | Baseline, 6 months and 12 months |
| New York |
| New York |
| 10032 |
| United States |
| Albert Einstein College of Medicine | The Bronx | New York | 10461 | United States |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| 31615480 | Background | Smaldone A, Manwani D, Aygun B, Smith-Whitley K, Jia H, Bruzzese JM, Findley S, Massei J, Green NS. HABIT efficacy and sustainability trial, a multi-center randomized controlled trial to improve hydroxyurea adherence in youth with sickle cell disease: a study protocol. BMC Pediatr. 2019 Oct 15;19(1):354. doi: 10.1186/s12887-019-1746-6. |
| 38321562 | Result | Green NS, Manwani D, Aygun B, Appiah-Kubi A, Smith-Whitley K, Castillo Y, Soriano L, Jia H, Smaldone AM. Hydroxyurea Adherence for Personal Best in Sickle Cell Treatment (HABIT) efficacy trial: Community health worker support may increase hydroxyurea adherence of youth with sickle cell disease. Pediatr Blood Cancer. 2024 Apr;71(4):e30878. doi: 10.1002/pbc.30878. Epub 2024 Feb 6. |
| 35614571 | Result | Green NS, Manwani D, Smith-Whitley K, Aygun B, Appiah-Kubi A, Smaldone AM. Mental health assessment of youth with sickle cell disease and their primary caregivers during the COVID-19 pandemic. Pediatr Blood Cancer. 2022 Sep;69(9):e29797. doi: 10.1002/pbc.29797. Epub 2022 May 25. |
| 39382077 | Derived | Lalji R, Koh L, Francis A, Khalid R, Guha C, Johnson DW, Wong G. Patient navigator programmes for children and adolescents with chronic diseases. Cochrane Database Syst Rev. 2024 Oct 9;10(10):CD014688. doi: 10.1002/14651858.CD014688.pub2. |
Dyads randomized to the control group will receive:
Standard care and Education handouts.
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| No other chronic condition besides Sickle Cell Disease (SCD) | Count of Participants | Participants |
|
| Had 1 or more ED visits in past year | Participants with one or more emergency department (ED) visits in the year prior to enrollment | Count of Participants | Participants |
|
| Had 1 more hospitalizations in past year | Participants with one or more hospitalizations in the year prior to enrollment | Count of Participants | Participants |
|
| Parent status: Single, separated or divorced | Count of Participants | Participants |
|
| Parent education high school or less | Count of Participants | Participants |
|
| Caregiver affected by SCD | Count of Participants | Participants |
|
| Other people in home affected by SCD | Count of Participants | Participants |
|
| Biomarker Fetal Hemoglobin (HbF) | Measure of the percentage of fetal hemoglobin in the blood | Mean | Standard Deviation | Percent |
|
| Proportion of days covered by hydroxyurea | The proportion of days covered by hydroxyurea in the year prior to study enrollment, using prescription refill data. | Mean | Standard Deviation | Percent of days |
|
Dyads randomized to the control group will receive: Standard care and Education handouts. |
|
|
|
| Primary | Mean Change in Proportion of Days Covered (PDC) by Hydroxyurea | The days covered by hydroxyurea was assessed using youth prescription refill data and was used to measure hydroxyurea adherence. The baseline measure is the proportion of days covered by hydroxyurea in the year prior to study enrollment, using prescription refill data. | 21 of 24 intervention group participants and 22 of 26 control group participants analyzed as data could not be collected from pharmacies due to COVID-19 difficulties and loss of data resulting from fire. | Posted | Mean | Standard Deviation | change in percentage of PDC | Baseline, 6 months, 12 months |
|
|
|
|
| Secondary | Mean Change in Youth Score on Peds Quality of Life (Generic Quality of Life) | Used to measure health-related quality of life. Scores range from 0-100 with a higher score indicating a better quality of life. | Posted | Mean | Standard Deviation | score on a scale | Baseline, 4 months, 9 months and 12 months |
|
|
|
|
| Secondary | Mean Change in Youth Score on PedsQL Sickle Cell Disease Module (Disease Specific Quality of Life) | Used to measure sickle cell disease specific health-related quality of life. Scores range from 0-100 with a higher score indicating a better quality of life. | Posted | Mean | Standard Deviation | score on a scale | Baseline, 9 months and 12 months |
|
|
|
|
| Secondary | Mean Change in Parent Youth Concordance Regarding Self-management Responsibility | Concordance between parent and youth scores Sickle Cell Family Responsibility scores for 11 items measuring self-management tasks. Scores range from 0-11 with a higher score indicating better concordance. | Posted | Mean | Standard Deviation | units on a scale | Baseline, 6 months and 12 months |
|
|
|
|
| 0 |
| 26 |
| 0 |
| 26 |
| 0 |
| 26 |
| EG001 | Intervention Group | In addition to standard care and education handouts, dyads randomized to the intervention group will receive the HABIT intervention, which includes CHW support and tailored text messages. | 0 | 24 | 0 | 24 | 0 | 24 |
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| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D010349 | Patient Compliance |
| D010342 | Patient Acceptance of Health Care |
| D000074822 | Treatment Adherence and Compliance |
| D015438 | Health Behavior |
| D001519 | Behavior |
| Regression, Linear |
| 0.04 |
| Non-Inferiority |
30% of the standard deviation was used as the non-inferiority margin. For this test generic quality of life (QOL) scores for months 4-9 (efficacy period) were compared to months >9 (sustainability period). |
| Regression, Linear |
| 0.045 |
| Non-Inferiority |
30% of the standard deviation was used as the non-inferiority margin. For this test sickle cell disease specific quality of life (SC-QOL) scores for months 4-9 (efficacy period) were compared to months >9 (sustainability period). |
| Regression, Linear |
| 0.23 |
| Non-Inferiority |
50% of the standard deviation was used as the non-inferiority margin. For this test youth caregiver concordance for self-management responsibility scores for months 0-6 (efficacy period) were compared to months 6-12 (sustainability period). |