Patient-reported outcomes in children with sickle cell disease at presentation for an acute pain episode

Bakshi, Nitya; Liu, Zihao; Gillespie, Scott; Keesari, Rohali; Leake, Deborah; Khemani, Kirshma; Kumari, Polly; Rees, Chris A.; Dampier, Carlton; Morris, Claudia R.

doi:10.1182/bloodadvances.2021006794

Cited by 2 publications

(2 citation statements)

References 22 publications

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“…55 Behavioral pain interventions are particularly important in addressing pain interference, as there is the variability of the extent to which even disease-modifying therapies (e.g., hydroxyurea) impact pain interference, if at all. 56,57 Even in studies where objective measures of hydroxyurea adherence were associated with worse pain interference, the difference between groups with high versus low adherence was approximately 1.4 SDs in patients with HbSS. 57 Comparatively, our behavioral intervention resulted in improvements in pain interference of approximately 2.4…”

Section: Discussionmentioning

confidence: 99%

“…Approaching the treatment of pain in SCD from a biopsychosocial rather than a biomedical perspective 54 is gaining momentum with increased appreciation for behavioral interventions that address the unique pain presentations seen in SCD 55 . Behavioral pain interventions are particularly important in addressing pain interference, as there is the variability of the extent to which even disease‐modifying therapies (e.g., hydroxyurea) impact pain interference, if at all 56,57 . Even in studies where objective measures of hydroxyurea adherence were associated with worse pain interference, the difference between groups with high versus low adherence was approximately 1.4 SDs in patients with HbSS 57 .…”

Section: Discussionmentioning

confidence: 99%

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Feasibility and preliminary efficacy of the Balance Program to reduce pain‐related disability in pediatric sickle cell disease

Connolly,

Forman,

Sharkey

et al. 2023

Pediatric Blood & Cancer

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BackgroundSickle cell disease (SCD) pain is associated with functional impairment, and treatment is often limited to pharmacological approaches with unwanted side effects. Although behavioral interventions exist for non‐SCD pain populations, interventions designed to address pain‐related impairment in SCD are lacking.MethodsTwenty youth (9‐17 years) with SCD completed a four‐week telemedicine pain intervention (NCT04388241). Participants and caregivers completed baseline and post‐intervention PROMIS pain measures and the Treatment Evaluation Inventory‐Short Form (TEI‐SF). Descriptive analyses assessed feasibility and acceptability. Reliable Change Index analyses assessed for significant post‐intervention changes in pain functioning. Paired t test analyses compared baseline and post‐intervention opioid prescription fills.ResultsAll participants completed at least one treatment session. Eighteen (90%) youth completed all sessions. Youth (100%) and caregivers (94%) rated the intervention as moderately or highly acceptable on the TEI‐SF. Forty‐seven percent of caregivers and 44% of youth reported reliably significant improvements in pain interference after the intervention (median T‐score differences: 24.8 and 23.5, respectively). Sixty‐five percent of caregivers (T‐score improvement difference: 19.3) and 31% of youth (T‐score improvement difference: 32) reported improvements in pain behaviors. There was no significant difference in opioid prescription fills pre‐ and post‐intervention (P > 0.05).ConclusionsThe Balance Program is feasible, highly acceptable, and can be delivered remotely to reduce barriers to care. Approximately half of youth and caregivers reported significant declines in pain interference following the intervention, with substantial improvements in functioning for treatment responders. Behavioral pain interventions are important adjunctive treatments to uniquely address functional impairment associated with acute and chronic pain in SCD.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Feasibility and preliminary efficacy of the Balance Program to reduce pain‐related disability in pediatric sickle cell disease

Connolly,

Forman,

Sharkey

et al. 2023

Pediatric Blood & Cancer

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Sickle Cell Disease Treatment with Arginine Therapy (STArT): study protocol for a phase 3 randomized controlled trial

Rees,

Brousseau,

Cohen

et al. 2023

Trials

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Background Despite substantial illness burden and healthcare utilization conferred by pain from vaso-occlusive episodes (VOE) in children with sickle cell disease (SCD), disease-modifying therapies to effectively treat SCD-VOE are lacking. The aim of the Sickle Cell Disease Treatment with Arginine Therapy (STArT) Trial is to provide definitive evidence regarding the efficacy of intravenous arginine as a treatment for acute SCD-VOE among children, adolescents, and young adults. Methods STArT is a double-blind, placebo-controlled, randomized, phase 3, multicenter trial of intravenous arginine therapy in 360 children, adolescents, and young adults who present with SCD-VOE. The STArT Trial is being conducted at 10 sites in the USA through the Pediatric Emergency Care Applied Research Network (PECARN). Enrollment began in 2021 and will continue for 5 years. Within 12 h of receiving their first dose of intravenous opioids, enrolled participants are randomized 1:1 to receive either (1) a one-time loading dose of L-arginine (200 mg/kg with a maximum of 20 g) administered intravenously followed by a standard dose of 100 mg/kg (maximum 10 g) three times a day or (2) a one-time placebo loading dose of normal saline followed by normal saline three times per day at equivalent volumes and duration as the study drug. Participants, research staff, and investigators are blinded to the participant’s randomization. All clinical care is provided in accordance with the institution-specific standard of care for SCD-VOE based on the 2014 National Heart, Lung, and Blood Institute guidelines. The primary outcome is time to SCD-VOE pain crisis resolution, defined as the time (in hours) from study drug delivery to the last dose of parenteral opioid delivery. Secondary outcomes include total parental opioid use and patient-reported outcomes. In addition, the trial will characterize alterations in the arginine metabolome and mitochondrial function in children with SCD-VOE. Discussion Building on the foundation of established relationships between emergency medicine providers and hematologists in a multicenter research network to ensure adequate participant accrual, the STArT Trial will provide definitive information about the efficacy of intravenous arginine for the treatment of SCD-VOE for children. Trial registration The STArT Trial was registered in ClinicalTrials.gov on April 9, 2021, and enrollment began on June 21, 2021 (NCT04839354).

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Patient-reported outcomes in children with sickle cell disease at presentation for an acute pain episode

Cited by 2 publications

References 22 publications

Feasibility and preliminary efficacy of the Balance Program to reduce pain‐related disability in pediatric sickle cell disease

Feasibility and preliminary efficacy of the Balance Program to reduce pain‐related disability in pediatric sickle cell disease

Sickle Cell Disease Treatment with Arginine Therapy (STArT): study protocol for a phase 3 randomized controlled trial

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