Background
Tricuspid regurgitation velocity (TRV), measured by echocardiography, is a surrogate marker for pulmonary hypertension. Limited pediatric studies have considered the association between ...TRV and surrogate markers of end‐organ disease.
Methods
We conducted a cross‐sectional study that evaluated the prevalence of elevated TRV ≥2.5 m/s and its associations with renal and cerebrovascular outcomes in children with sickle cell disease (SCD) 1–21 years of age in two large sickle cell cohorts, the University of Alabama at Birmingham (UAB) sickle cell cohort, and the Sickle Cell Clinical Research and Intervention Program (SCCRIP) cohort at St. Jude Children's Research Hospital. We hypothesized that patients with SCD and elevated TRV would have higher odds of having either persistent albuminuria or cerebrovascular disease.
Results
We identified 166 children from the UAB cohort (mean age: 13.49 ± 4.47 years) and 325 children from the SCCRIP cohort (mean age: 13.41 ± 3.99 years) with echocardiograms. The prevalence of an elevated TRV was 21% in both UAB and SCCRIP cohorts. Elevated TRV was significantly associated with cerebrovascular disease (odds ratio OR 1.88, 95% confidence interval CI: 1.12–3.15; p = .017) and persistent albuminuria (OR 1.81, 95% CI: 1.07–3.06; p = .028) after adjusting for age, sex, treatment, and site.
Conclusion
This cross‐sectional, multicenter study identifies associations between surrogate markers of pulmonary hypertension with kidney disease and cerebrovascular disease. A prospective study should be performed to evaluate the longitudinal outcomes for patients with multiple surrogate markers of end‐organ disease.
Summary
Neurocognitive impairment is common in sickle cell disease (SCD) and is associated with significant functional limitations. In a cross‐sectional analysis, we examined the association between ...hydroxyurea (HU) treatment and neurocognitive functioning from school‐age to young adulthood in individuals with SCD. A total of 215 patients with HbSS/HbSβ0‐thalassaemia (71% HU treated) and 149 patients with HbSC/HbSβ+‐thalassaemia (20% HU treated) completed neurocognitive measures at one of four developmental stages: school‐age (age 8–9 years), early adolescence (age 12–13 years), late adolescence (age 16–17 years) and young adulthood (ages 19–24 years). For participants with multiple assessments, only the most recent evaluation was included. In multivariable analysis adjusted for social vulnerability, HU treatment and sex, older age was associated with a reduction in overall intelligence quotient (IQ) of 0·55 points per year of life standard error (SE) = 0·18, false discovery rate adjusted P value (PFDR) = 0.01 for patients with HbSS/HbSβ0‐thalassaemia. Earlier initiation of HU (n = 152) in HbSS/HbSβ0‐thalassaemia was associated with higher scores on neurocognitive measures across most domains, including IQ estimate (SE) 0·77 (0·25)/year, PFDR = 0·01, after adjusting for social vulnerability, sex and treatment duration. These results support the early use of HU to limit the detrimental neurocognitive effects of SCD, while highlighting the need for additional measures to further mitigate neurocognitive deterioration.
Pain is a primary symptom of sickle cell disease (SCD) and is often severe and chronic. To treat SCD-related pain, proper assessment of SCD pain among youth, including the degree of concordance or ...agreement between youth and caregiver reports of pain, is essential but has not yet been adequately evaluated. In this study, 525 youth with SCD and their parents were evaluated as part of the Sickle Cell Clinical Research and Intervention Program (SCCRIP) to examine pain rating concordance and predictors of concordance. Youth and parents completed the Pediatric Quality of Life Inventory Sickle Cell Disease module (PedsQL-SCD) to measure pain, pain interference, and pain-related constructs. Disease, clinical, and demographic variables were obtained from the SCCRIP database. Intraclass correlations demonstrated moderate-to-poor consistency between youth and caregiver reports of pain and pain interference (ICCs range from 0.17 to 0.54). Analysis of covariance and regression models found that patient age, frequency of hospitalizations and emergency department (ED) visits, economic hardship, and fetal hemoglobin levels were significantly associated with varying pain-rating agreement levels among parent proxy and child self-report pain. Concordance of pain assessments among youth with SCD and their caregivers using the PedsQL-SCD Module was moderate at best, corroborating prior research. Youth factors predicting discordance among pain-related factors included increased ED visits, older age, and female sex. Collectively, these results bolster the use of integrated pain assessments to reduce parent-child discrepancies, thereby improving the adequacy of SCD-related pain assessment and treatment.
Background
Immunosuppressive therapy with horse antithymocyte globulin and cyclosporine currently remains the standard therapy for children with severe aplastic anemia (SAA) who lack human leukocyte ...antigen (HLA)‐identical sibling. The thrombopoietin receptor agonist eltrombopag has been recently approved for SAA patients 2 years and older. However, there are limited data on its safety and efficacy in pediatric cohorts.
Methods
We conducted a retrospective study of patients ≤18 years old consecutively diagnosed with SAA between 2000 and 2018. Patients received either standard immunosuppressive therapy (IST‐Std) or IST with eltrombopag (IST‐Epag). The primary outcome was the objective response (OR), including partial and complete response (CR), at 6 and 12 months after starting therapy.
Results
We identified 16 patients receiving IST‐Std and nine IST‐Epag treatment (seven of nine as upfront therapy and two of seven after previously failed IST). The OR at 6 and 12 months in IST‐Std arm was 71% and 100%, with CR in 29% and 58%, respectively. Seven patients receiving upfront IST‐Epag had OR at 6 and 12 months, with two of seven (29%) achieving CR at 6 and 12 months. Two patients who previously failed standard IST did not respond to eltrombopag. No significant differences were observed in both cohorts with regard to infections. One IST‐Epag‐treated patient developed transient grade 3 transaminitis. Finally, no changes in paroxysmal nocturnal hemoglobinuria (PNH) clone size and cytogenetic abnormalities were seen in either cohort.
Conclusion
The addition of eltrombopag to standard IST was well tolerated and resulted in satisfactory hematological response at 6 and 12 months in this single‐institution experience. A larger cohort with longer follow‐up is required to assess response durability.
To evaluate the effectiveness of a vaccine strategy bundle to increase human papillomavirus (HPV) vaccine initiation and completion in a specialty clinic setting.
Our Hematology clinic utilized an ...implementation framework from October 1, 2018, to December 31, 2019, involving nurses, nursing coordinators, and clinicians in administering the HPV vaccination series to our adolescent sickle cell sample of nearly 500 patients. The bundle included education for staff on the need for HPV vaccine administration, provider incentives, vaccines offered to patients in SCD clinics, and verification of patients' charts of vaccine completion.
Following the implementation of the bundle, the cumulative incidence of HPV vaccination initiation and completion improved from 28% to 46% and 7% to 49%, respectively. Both rates remained higher postimplementation as well. HPV vaccination series completion was associated with a decreased distance to the health care facility, lower state deprivation rank, and increased hospitalizations.
Our clinic's implementation strategy successfully improved vaccine completion rates among adolescents with sickle cell disease (SCD) while continuing to educate staff, patients, and families on the importance of cancer prevention among people living with SCD.
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