Hydroxyurea use in young infants with sickle cell disease.

BACKGROUND: Hydroxyurea (HU) reduces complications and improves quality and duration of life in sickle cell disease. Evidence supports the use of HU starting after nine months of age. PROCEDURES: We performed a retrospective study of patients starting HU at less than five years of age between January 1, 2008, and December 31, 2016. We evaluated clinical events, laboratory data, and toxicity between three different age groups: cohort 1 (0-1 year), cohort 2 (1-2 years), and cohort 3 (2-5 years). RESULTS: Sixty-five patients were included in the analysis. The mean age was 7.2 months (n = 35), 19.5 months (n = 13), and 35.5 months (n = 17) for cohorts 1, 2, and 3, respectively. Cohort 1 had higher hemoglobin (P = 0.0003) and MCV (P = 0.0199) and lower absolute reticulocyte count (P = 0.0304) at 24 months of age compared with cohort 3. The absolute neutrophil count (ANC) was lower compared with both older cohorts (P = 0.0364, 0.0025). The mean baseline hemoglobin F in cohort 1 was 31.5% compared with 19.7% and 16.5% in cohorts 2 and 3, respectively (P = 0.002, P < 0.0001). The mean duration of therapy was 31.3 months, 57.6 months (P = 0.018), and 29.1 months (P = 0.401), respectively. Mean Hb F levels remained higher in cohort 1 (29.9%) compared with cohorts 2 and 3 (20.4%, P = 0.007; 20.6%, P = 0.003). Cohort 1 experienced fewer hospitalizations (P = 0.0025), pain crises (P = 0.0618), and transfusions (P = 0.0426). There was no difference in toxicity between groups. CONCLUSION: HU is safe and effective in patients 5 to 12 months of age and generated a more robust response compared with initiation in older patients.

Invasive pneumococcal disease in children with sickle cell disease in the pneumococcal conjugate vaccine era.

Invasive pneumococcal disease (IPD) in children with sickle cell disease (SCD) can be devastating. We sought to assess the impact of IPD in children with SCD since licensure of the pneumococcal conjugate vaccines (PCVs). We found 11 cases of IPD giving an incidence of 417 per 100,000 person-years, much higher than that reported in children without SCD. Although all isolates were sensitive to penicillin, 89% of isolates were nonvaccine serotypes. Further study is needed to characterize the incidence of and risk factors for the development of IPD in SCD in the PCV era to help drive better prevention strategies.

Incorporating integrative medicine and patient preferences into a pilot interdisciplinary sickle cell wellness clinic.

OBJECTIVE: Sickle Cell Disease (SCD) is an inherited blood disorder that includes acute pain episodes and chronic pain that can dramatically impact quality of life and goal-achievement. Our staff had limited success in connecting families with the Pain, Palliative Care and Integrative Medicine Clinic (PPCIM) to receive specialized skills for pain management. We created a partnership between Hematology and PPCIM to provide SCD patients/families with needed resources. DESIGN/SETTING: In 2016, key stakeholders collaborated to create a Sickle Cell Wellness Clinic (SCWC) clinic to provide families access to integrative medicine and wellness strategies. Design/structure, based on family focus group data and staff expertise, included a half-day, 7-discipline clinic housed in the PPCIM space. Patients with SCD, ages 8-20, learned strategies in an effort to improve health care utilization and increase overall quality of life. MAIN OUTCOME MEASURES/RESULTS: Feedback from two successful pilot clinics in 2017 was incorporated into the formal roll-out of SCWC in 2018. SCWCs continued monthly for one year, serving a total of 20 families post-pilot. SCD patients increased follow-up appointment engagement in the PPCIM clinic following SCWC and reported high levels of satisfaction with their healthcare experience. CONCLUSIONS: It is feasible to run a multidisciplinary clinic focused on pain management, coping skills, and healthy living with SCD. Providers benefited from the opportunity to collaborate with other disciplines. Patient and family feedback was positive, highlighted benefits of being introduced to new modalities, and reported advantages of meeting other patients/families in a new setting.

High prevalence of pulmonary hypertension in children with sickle cell disease.

Pulmonary hypertension (PHT) affects approximately 30% of adults with sickle cell disease. Adults with PHT have a significantly higher mortality rate. We report the results of a prospective study of the prevalence of PHT among children with sickle cell disease. In our cohort, 31% of children>or=10 years of age have evidence of PHT by Doppler echocardiography. Factors associated with the presence of PHT are male sex and elevated reticulocyte count. We recommend screening all children for PHT starting at the age of 10 years.