Alprostadil
| Evidence Level: L5 | Predicted Indications: 9 |
Table of Contents
Alprostadil: Aortic malformation
One-Sentence Summary
Alprostadil is a prostaglandin E1 analogue that relaxes smooth muscle and keeps the ductus arteriosus open. The TxGNN model predicts it may be useful for aortic malformation, with a prediction score of 0.9998. The data compiled for this report contain no registered clinical trials and 8 publications (mostly case reports, cohort studies and one perioperative RCT) for this direction.
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Not recorded in the local data |
| Predicted New Indication | Aortic malformation |
| TxGNN Prediction Score | 0.9998 |
| Evidence Level | L5 |
| Australia Market Status | Listed on the PBS Schedule (1 of 1 items) – TGA (ARTG) registration status is not included in this data |
| Number of PBS Items | 1 (valid: 1) |
| Recommended Decision | Hold |
Why is This Prediction Reasonable?
Detailed mechanism of action data are not available in the data compiled for this report. Alprostadil is prostaglandin E1 (PGE1). Its receptor-mediated effect is well established: it relaxes the smooth muscle of the ductus arteriosus and keeps the duct open.
This matters for aortic lesions in which the systemic circulation depends on a patent duct, such as coarctation of the aorta and interrupted aortic arch. In these neonates, alprostadil acts as a temporising bridge until surgical repair. The mechanistic link is therefore plausible, but it supports a bridging role rather than treatment of the malformation itself.
The evidence level is L5 because none of the cited studies directly studies aortic malformation as a defined disease. The studies concern congenital heart disease in general or individual lesions (for example interrupted aortic arch in a single case report). They are described below as supportive context only.
Clinical Trial Evidence
Currently no related clinical trials registered.
Literature Evidence
| PMID | Year | Type | Journal | Key Findings |
|---|---|---|---|---|
| 19080093 | 2008 | RCT | Zhonghua Yi Xue Za Zhi | Alprostadil and ulinastatin studied for inflammatory response and lung injury after cardiopulmonary bypass in children with congenital heart disease |
| 6763200 | 1982 | Cohort | Pharmacotherapy | Evaluation of alprostadil in infants with congenital heart disease; it dilates the ductus and increases pulmonary blood flow in duct-dependent lesions |
| 15362549 | 2004 | Review | Kyobu Geka | Transposition of the great arteries: alprostadil keeps the ductus patent to protect left ventricular performance before arterial switch surgery |
| 41167776 | 2025 | Cohort | Zhonghua Fu Chan Ke Za Zhi | Retrospective study of delivery mode and timing in 123 fetuses with complex congenital heart disease |
| 42469655 | 2026 | Cohort | BMC Pregnancy Childbirth | Retrospective study of pregnancy and perinatal management of complex fetal congenital heart disease |
| 19068708 | 2008 | Case report | Kyobu Geka | Neonate with interrupted aortic arch type B kept ductus open with alprostadil, then survived single-stage repair with haemodiafiltration |
| 30184041 | 2018 | Case report | Braz J Cardiovasc Surg | Aortopulmonary window repair in a 9-year-old; milrinone and alprostadil used before surgery to counter high lung pressure |
| 20029353 | 2009 | Preclinical | Minerva Chirurgica | Alprostadil reduced ischaemia-reperfusion injury in rat skeletal muscle (not specific to aortic disease) |
Australia Market Information
| ARTG Number | Product Name | Dosage Form | Approved Indication |
|---|---|---|---|
| ARTG 4579B | Caverject Impulse | Intracavernosal injection 10 micrograms with diluent in single use syringe | Not stated in the data compiled |
Safety Considerations
- Contraindications: Label-based contraindication data are not available in the data compiled for this report. Please refer to the TGA-approved Product Information (PI).
- Reported safety signals in the literature: Prolonged prostaglandin E1 infusion in neonates has been linked to pseudo-Bartter syndrome and to gastric outlet obstruction (antral foveolar hyperplasia or hypertrophic pyloric stenosis).
- Drug Interactions: 59 interactions were identified. Moderate-level interactions include canagliflozin, dapagliflozin, empagliflozin, ertugliflozin, lorcaserin and sapropterin. Others, with an unclassified severity level, include morphine, metformin, omeprazole, pantoprazole, prednisolone, prednisone, simvastatin, vancomycin, atropine and amphotericin B.
- Note on the local product: The only Australian licence is an intracavernosal injection. It is not a neonatal intravenous infusion product, so route and formulation compatibility with the predicted use has not been established.
Conclusion and Next Steps
Decision: Hold
Rationale: The prediction score is high and the mechanism for duct-dependent aortic lesions is plausible. However, no cited study directly addresses aortic malformation as a defined disease, there are no registered trials, and the only Australian product is an intracavernosal formulation.
To proceed, the following is needed:
- Studies or trials that directly address aortic malformation or specific duct-dependent aortic lesions
- Confirmation of an appropriate neonatal intravenous formulation and its regulatory status in Australia
- Mechanism of action data and label-based warnings and contraindications from the TGA Product Information
- A neonatal safety monitoring plan, including gastric outlet obstruction and electrolyte disturbance during prolonged infusion
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.