Last reviewed 2026-04-20 · How we verify

THIOTEPA

At a glance

Approved indications

No approved indications tracked.

Common side effects

• Neutropenia
• Anemia
• Thrombocytopenia
• Elevated alanine aminotransferase
• Elevated aspartate aminotransferase
• Elevated bilirubin
• Mucositis
• Cytomegalovirus infection
• Hemorrhage
• Diarrhea
• Hematuria
• Rash

Serious adverse events

• Veno-occlusive disease
• Gastrointestinal hemorrhage
• Pneumonia
• Seizure
• Subarachnoid hemorrhage

Key clinical trials

• Dinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma (PHASE3)
• Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases (PHASE1, PHASE2)
• IT-TT as an Effective and Well-Tolerated Strategy for CNS Prop in High-Risk DLBCL: a Prospective Ph II Study (PHASE2)
• US Study of ECT-001-CB in Pediatric and Young Adult Patients With High-Risk Myeloid Malignancies (PHASE1, PHASE2)
• US Study of UM171-Expanded CB in Patients With High Risk Leukemia/Myelodysplasia (PHASE2)
• ASCT With TEAM Conditioning for Lymphoma With High Risk of CNS Relapse (PHASE2)
• Trial for Patients With Newly Diagnosed Primary Central Nervous System (CNS) Lymphoma (PHASE2)
• OptiMATe: De-escalated Induction Treatment in Primary CNS Lymphoma (PHASE3)

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• THIOTEPA CI brief — competitive landscape report
• THIOTEPA updates RSS · CI watch RSS