Longitudinal follow up of elevated pulmonary artery pressures in children with sickle cell disease

Abstract

Summary Elevated pulmonary artery pressures (PAP) occur in approximately 30% of children with sickle cell disease. In adults, pulmonary hypertension is significantly associated with mortality. There are no data on the long term significance in children. Nineteen children with SS/Sß0 thalassaemia had elevated PAP, defined as tricuspid regurgitant jet velocity (TRV) ≥2·5 m/s on screening echocardiograms. They were prospectively followed for 23 months (range 19–31 months). Patients with initial TRV ≥ 3 or TRV ≥ 2·5 m/s on repeat echocardiogram had cardiopulmonary evaluation and were offered treatment with hydroxyurea. Associated conditions like asthma and obstructive sleep apnea were treated. 18/19 patients had follow‐up echocardiograms. These showed normalization of TRV in 8 patients. Risk factors associated with persistent elevation were higher TRV on initial echocardiogram (P = 0·01), lower haemoglobin (P = 0·003) and lower oxygen saturation (P = 0·03). Five patients with persistently elevated PAP were treated with hydroxyurea. Mean right ventricular pressure dropped from 40·16 to 29·26 (P = 0·017) after 3–6 months and to 23·6 mmHg (P = 0·002) after 9–12 months on treatment. In conclusion (i) At borderline elevation of TRV there is intrapatient variability and echocardiograms should be repeated for confirmation. (ii) Elevated PAP are reversible in children with early detection and treatment with hydroxyurea.

An Official American Thoracic Society Clinical Practice Guideline: Diagnosis, Risk Stratification, and Management of Pulmonary Hypertension of Sickle Cell Disease

“…The final judgment of the committee was to compromise; that is, to apply the evidence in our decision making, but to lower the quality of evidence to reflect the indirectness of the population. The case series support this extrapolation of evidence, finding that HU treatment of patients with SCD with elevated TRV produces a decline in TRV (31,32). This occurred most often in children and young adults with a mild TRV elevation in whom HU increased the fetal hemoglobin to more than 20%.…”

Relationship of erythropoietin, fetal hemoglobin, and hydroxyurea treatment to tricuspid regurgitation velocity in children with sickle cell disease

“…Consistent with this possibility, a recent publication reported reduction in mean pulmonary artery pressures with hydroxyurea therapy in 5 patients. 38 On the other hand, prospective administration of hydroxyurea in the Multicenter Study of Hydroxyurea in Sickle Cell Anemia did not influence concentrations of N-terminal pro-brain natriuretic peptide, an index of pulmonary hypertension. 10 There are several additional limitations to our study.…”

Hemodynamic Predictors of Mortality in Adults with Sickle Cell Disease

“…A much larger number of therapies approved for PH in the general population have become available over the intervening years, potentially permitting better PH control. Last, supportive care of SCD with hydroxyurea or chronic transfusion has been more aggressively promoted in the last decade, and anecdotal evidence indicates that in some cases this may lead to better control of pulmonary arterial pressures (39,40).…”