Praziquantel

證據等級: L5

預測適應症: 10 個

Praziquantel: From Schistosomiasis to Plasmodium falciparum Malaria

One-Sentence Summary

Praziquantel is the standard antiparasitic agent used to treat schistosomiasis (bilharzia) — a use confirmed repeatedly within this evidence pack’s own trial and literature records, even though formal regulatory indication text is not available. TxGNN’s single highest-scoring prediction is a cluster of soft-tissue sarcomas (leiomyosarcoma-type diagnoses) that carries no clinical trial or literature support and no plausible mechanistic link to an antiparasitic drug. The next-ranked, evidence-backed prediction — Plasmodium falciparum malaria — is supported by 5 clinical trials and 20 publications, including a 2026 double-blind, placebo-controlled randomized trial testing praziquantel directly against P. falciparum infection.

Quick Overview

Item	Content
Original Indication	Schistosomiasis (inferred from clinical-trial and literature references in this pack; formal SAHPRA-approved indication text is a data gap — DG001)
Predicted New Indication	Plasmodium falciparum malaria
TxGNN Prediction Score	97.22% (rank 11,228 of all drug–disease pairs)
Evidence Level	L2
South Africa Market Status	Not marketed
Number of SAHPRA Registrations	0
Recommended Decision	Hold

Why is This Prediction Reasonable?

Currently, detailed mechanism of action data is not available (DG002 — MOA is a documented data gap in this evidence pack). Based on known information within the evidence pack itself, praziquantel is repeatedly described as the mainstay anthelmintic for schistosomiasis (e.g., “Praziquantel (PZQ) remains the sole effective drug against schistosomiasis,” PMID 37446846; the SchistoSAM trial NCT03893097 benchmarks a new regimen against “standard praziquantel (PZQ) treatment”).

It is worth noting that TxGNN’s single top-ranked prediction for this drug (uterine corpus epithelioid leiomyosarcoma, score 97.28%) and several closely-scored neighbours (retroperitoneal sarcoma, uterine myxoid leiomyosarcoma, anus leiomyosarcoma, small intestinal sarcoma, leiomyosarcoma) return zero clinical trials and zero literature hits, and there is no known pharmacological rationale connecting an antiparasitic agent to soft-tissue sarcoma biology. This pattern is most consistent with a knowledge-graph embedding artifact rather than a genuine repurposing signal, and it is not carried forward as the headline finding of this report.

By contrast, malaria and other parasitic diseases (fascioliasis, gnathostomiasis) cluster just below the sarcoma group in TxGNN score and are mechanistically coherent: praziquantel’s antiparasitic activity against trematodes/cestodes plausibly extends to other parasite life-cycle targets, and this overlap is directly reflected in real-world co-endemic research (schistosomiasis and malaria co-occur across sub-Saharan Africa, and mass drug administration programmes already combine anthelmintic and antimalarial treatment). The strongest single piece of evidence is a purpose-built 2026 RCT (PMID 41159886) directly testing praziquantel’s antimalarial efficacy in P. falciparum-infected adults.

Clinical Trial Evidence

(For Plasmodium falciparum malaria; note most of these trials study malaria–helminth co-infection populations where praziquantel is used for deworming, with malaria as a co-measured outcome, rather than dedicated praziquantel-vs-malaria registration trials.)

Trial Number	Phase	Status	Enrollment	Key Findings
NCT03640403	Phase 3	Completed	1,555	Intermittent preventive treatment trial in school-aged children in Tanzania, in a population where malaria/helminth co-infection and cognitive impact are studied together.
NCT01722539	Phase 3	Completed	616	Intermittent preventive antimalarial regimens in DRC schoolchildren; relevant as a co-infection control population where anthelmintic treatment status is tracked.
NCT03893097	Phase 3	Completed	726	Head-to-head trial of artesunate-mefloquine vs. standard praziquantel for schistosomiasis in a malaria-endemic Senegalese cohort.
NCT02769013	N/A	Completed	380	Assesses effect of neglected tropical diseases (incl. helminths treated with praziquantel) on P. falciparum transmission dynamics.
NCT00347113	N/A	Completed	620	Anthelminthic intervention trial evaluating effect on malaria infection/morbidity in Tanzanian school and pre-school children.

Literature Evidence

PMID	Year	Type	Journal	Key Findings
41159886	2026	RCT	The Journal of Infectious Diseases	Double-blind, placebo-controlled RCT of praziquantel for P. falciparum infection in asymptomatic Gabonese adults — the direct efficacy evidence behind this prediction.
10531774	1998	Clinical study	JPMA (Pakistan Medical Association)	Small case series (9 P. falciparum, 1 P. vivax) treated with oral praziquantel 30 mg/kg/day; 8/10 achieved complete cure within 4–6 days.
37957702	2023	RCT	Malaria Journal	Randomized, observer-blind trial integrating helminth mass drug administration (praziquantel) with seasonal malaria chemoprevention in Senegalese children; feasible and safe.
25887977	2015	RCT (open-label)	BMC Infectious Diseases	Anthelmintic treatment approaches assessed for impact on malaria infection and anaemia in Tanzanian school/pre-school children.
21696629	2011	Intervention study	BMC Int Health & Human Rights	33-month follow-up of integrated school-based deworming plus prompt malaria treatment on helminth–P. falciparum co-infection.
38265982	2024	Scoping review	PLoS Neglected Tropical Diseases	Reviews prior efforts to integrate malaria and schistosomiasis prevention/control programmes.
20350194	2010	RCT (exploratory, open-label)	Clinical Infectious Diseases	Compares mefloquine, artesunate, mefloquine-artesunate and praziquantel efficacy/safety against Schistosoma haematobium in a malaria-endemic setting.
30080853	2018	Cohort study	PLoS Neglected Tropical Diseases	Schistosomiasis (praziquantel-treated) modifies P. falciparum infection prevalence/incidence in Gabonese school-age children.
25210876	2014	Prospective cohort	PLoS Neglected Tropical Diseases	Long-term P. falciparum carriers co-infected with S. haematobium show enhanced protection from febrile malaria in Mali.
24609234	2014	Review	Parasitology Research	Reviews dihydroartemisinin’s dual antimalarial/antischistosomal activity, contextualising drug repurposing across these two parasitic diseases.

South Africa Market Information

No SAHPRA product registrations are present in this evidence pack (total_licenses: 0, market status: not marketed). No registration number, product name, dosage form, or approved indication text is currently available for praziquantel in South Africa within the data reviewed.

Safety Considerations

Please refer to the SAHPRA-approved Professional Information (PI) for safety information. Report adverse drug reactions to SAHPRA.

(Key warnings, contraindications, and drug–drug interaction data were queried but returned no results in this evidence pack — flagged as a Blocking data gap, DG001.)

Conclusion and Next Steps

Decision: Hold

Rationale: Praziquantel is not currently marketed or SAHPRA-registered in South Africa, and mandatory safety data (PI warnings/contraindications) is a Blocking data gap (DG001) that prevents any S1 safety assessment. While the Plasmodium falciparum malaria signal is genuinely evidence-backed — including a 2026 placebo-controlled RCT — it stands alongside a top-ranked TxGNN prediction (leiomyosarcoma/sarcoma cluster) that has no supporting evidence and no plausible mechanism, indicating the raw model ranking should not be used to prioritize indications for this candidate without human review.

To proceed, the following is needed:

SAHPRA-approved PI (warnings, contraindications, DDI) to close DG001 before any safety evaluation
Confirmed mechanism of action data to close DG002 and support a mechanistic case for the malaria indication
Determination of whether SAHPRA registration/import pathway exists for praziquantel in South Africa
A confirmatory, adequately powered RCT of praziquantel specifically for P. falciparum malaria (current evidence is one small RCT plus mostly co-infection/observational studies)
Independent review confirming the leiomyosarcoma/sarcoma cluster of predictions should be deprioritized given the absence of any corroborating evidence or mechanistic rationale
Disclaimer

This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.