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A pilot trial testing the feasibility of administering D-penicillamine to extremely low birth weight neonates
R D Christensen1, S C Alder, S C Richards
1Department of Women and Newborn, Intermountain Health Care and the McKay-Dee Hospital Center, Ogden, UT 84403, USA. rdchris4@ihc.com
Insights
Enteral 3-mercapto-D-valine (D-penicillamine) was safely administered to extremely low birth weight (ELBW) neonates. This suggests potential for reducing retinopathy of prematurity (ROP) and warrants further safety and efficacy trials.
Area of Science:
- Neonatal Medicine
- Pharmacology
- Ophthalmology
Background:
- Retinopathy of prematurity (ROP) is a significant cause of visual impairment in extremely low birth weight (ELBW) neonates.
- Early therapeutic interventions are crucial for managing ROP and improving neonatal outcomes.
Purpose of the Study:
- To assess the feasibility and tolerability of enteral 3-mercapto-D-valine (D-penicillamine) administration in ELBW neonates.
- To explore the preliminary efficacy of D-penicillamine in reducing the incidence or severity of ROP.
Main Methods:
- A 14-day course of enteral D-penicillamine was administered to five ELBW neonates via nasogastric tube.
- Dosage adjusted over the treatment period, with daily monitoring for immediate intolerance.
- Laboratory tests evaluated hepatic, renal, and hematologic toxicity; ROP was scored using ICROP guidelines and compared to a historical cohort.
Main Results:
- All five neonates completed the full D-penicillamine course without immediate intolerance.
- No significant increase in adverse events (creatinine elevation, thrombocytopenia, neutropenia, hyperbilirubinemia, abnormal liver function) compared to the cohort.
- Only one neonate developed transient stage 1 ROP, compared to 54% in the control group.
Conclusions:
- Enteral administration of D-penicillamine is feasible and well-tolerated in ELBW neonates.
- Preliminary findings suggest a potential protective effect against ROP.
- Results support proceeding to Phase II trials for safety and efficacy evaluation.
Objective:
We enterally administered a 14-day course of 3-mercapto-D-valine (D-penicillamine) to five extremely low birth weight (ELBW) neonates, as a step toward assessing this therapy as a means of reducing the incidence or severity of retinopathy of prematurity (ROP).
Methods:
The study drug (100 mg/ml) was given by nasogastric tube at a dose of 100 mg/k every 8 h for three days, and then 50 mg/k once per day for 11 additional days. Logbooks were maintained by the bedside nurses to record signs of possible immediate intolerance. Laboratory tests assessed hepatic, renal, and hematologic toxicity. ROP was scored according to the ICROP guidelines. Comparisons were with a cohort of 139 consecutive recent neonates of the same birth weight and gestational age range.
Results:
Five neonates were enrolled in the study, and all received the full course of study drug as planned. Signs of immediate intolerance of the study drug were not observed in any. The study patients did not have a higher incidence, than that of the cohort group, in creatinine elevation, thrombocytopenia, neutropenia, hyperbilirubinemia, or abnormal liver function test. Four of the five had no ROP and one developed transient stage 1, compared with a 54% occurrence of ROP in the cohort.
Conclusions:
It is feasible to enterally administer a 14-day course of 3-mercapto-D-valine to ELBW neonates and the suspension appears to be well tolerated. These results suggest that phase II safety and preliminary efficacy trials can be undertaken.