Clinical Study to Evaluate the Possible Efficacy and Safety of L-Arginine in Children with Sickle Cell Disease and Increased Tricuspid Regurgitant Jet Velocity: a Randomized Controlled Trial.
- 2025-06-17
- Paediatric drugs 27(5)
- Dalia A Gomaa
- Sahar M El-Haggar
- Mohamed R El-Shanshory
- Osama El-Razaky
- Dalia R El-Afify
- PubMed: 40526198
- DOI: 10.1007/s40272-025-00701-w
Study Design
- Type
- Randomized Controlled Trial (RCT)
- Sample size
- n = 50
- Population
- 50 children with SCD who had TRJV higher than 2.5 m/s
- Methods
- randomly divided into two groups, each with 25 patients: group 1 (control group) and group 2 (treatment group). Group 2 received L-arginine at a dose of 0.1-0.2 g/kg/day for 3 months. Transthoracic echocardiography and blood samples were conducted at baseline and after 3 months
- Duration
- 3 months
Background
Pulmonary hypertension (PH) is a common chronic complication of sickle cell disease (SCD), and patients at risk for PH can be identified by measuring tricuspid regurgitant jet velocity (TRJV). We looked for the possible efficacy of L-arginine for children with SCD who have elevated TRJV.Methods
In total, 50 children with SCD who had TRJV higher than 2.5 m/s were randomly divided into two groups, each with 25 patients: group 1 (control group) and group 2 (treatment group). Group 2 received L-arginine at a dose of 0.1-0.2 g/kg/day for 3 months. Transthoracic echocardiography was conducted to measure TRJV at baseline and after 3 months, and blood samples were collected at baseline and after 3 months to assess serum levels of N-terminal pro-brain natriuretic peptide (NT-proBNP), lactate dehydrogenase (LDH), nitric oxide (NO), L-arginine (LA), asymmetric dimethylarginine (ADMA), and LA/ADMA ratio.Results
After 3 months of treatment, the L-arginine-treated group had significantly lower TRJV levels than the control group, and compared with baseline. They also had significantly lower NT-proBNP and significantly higher NO, LA and LA/ADMA ratios than the control group and compared with baseline. No significant differences in side effects were observed between the two groups, indicating that L-arginine is safe for these patients.Conclusions
L-arginine is associated with a reduction in TRJV, NT-proBNP, and improvements in NO biomarkers, suggesting it may be beneficial for reducing the risk of pulmonary hypertension in children with sickle cell disease.Clinical trial registration
ClinicalTrials.gov identifier NCT05470998.Research Insights
significantly higher ... LA
- Effect
- Beneficial
- Effect size
- Small
- Dose
- 0.1-0.2 g/kg/day
significantly higher ... LA/ADMA ratios
- Effect
- Beneficial
- Effect size
- Small
- Dose
- 0.1-0.2 g/kg/day
significantly higher NO
- Effect
- Beneficial
- Effect size
- Small
- Dose
- 0.1-0.2 g/kg/day
The abstract reports that LDH levels were assessed at baseline and after 3 months, but no significant difference between groups was explicitly mentioned in the results summary for LDH.
- Effect
- Neutral
- Effect size
- Small
- Dose
- 0.1-0.2 g/kg/day
They also had significantly lower NT-proBNP ... than the control group and compared with baseline.
- Effect
- Beneficial
- Effect size
- Moderate
- Dose
- 0.1-0.2 g/kg/day
They also had significantly lower NT-proBNP
- Effect
- Beneficial
- Effect size
- Small
- Dose
- 0.1-0.2 g/kg/day
After 3 months of treatment, the L-arginine-treated group had significantly lower TRJV levels than the control group
- Effect
- Beneficial
- Effect size
- Small
- Dose
- 0.1-0.2 g/kg/day
Adverse Events Reported
No significant differences in side effects were observed between the two groups, indicating that L-arginine is safe for these patients.
- Finding
- No significant difference
No significant differences in side effects were observed between the two groups, indicating that L-arginine is safe for these patients.
- Finding
- No significant difference
- Significant
- No