# Thiotepa

Source: https://onco.cc/drugs/thiotepa/  
OnCo record `thiotepa` (Treatment). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Thiotepa is an old chemotherapy with a modern job: at high doses it prepares patients for stem cell transplants, and it is still instilled into the bladder or body cavities for superficial bladder cancer and malignant effusions.

## Summary

Thiotepa was first approved in 1959 for adenocarcinoma of the breast or ovary, superficial papillary carcinoma of the urinary bladder and control of malignant intracavitary effusions. Tepadina (approved 2017 in the US, 2010 in the EU) added conditioning before allogeneic haematopoietic stem cell transplantation, initially for children with class 3 beta-thalassaemia in the US and, in the EU, for haematological and solid tumours in adults and children. In practice thiotepa-based regimens (TBC, thiotepa-busulfan-fludarabine) are standard before autologous transplant in primary CNS lymphoma and in cord blood and haploidentical transplantation. Profound myelosuppression, mucositis and skin toxicity from sweat excretion (patients bathe frequently) are the main issues.

## Fields

- Kind: Treatment
- Status: approved
- Last checked: 2026-09-10
- Also known as: Thioplex (historic); Triethylenethiophosphoramide
- Tags: nci-list
- Brand: Tepadina
- Modality: Alkylating agent (ethylenimine)
- Mechanism: Polyfunctional alkylating agent related to nitrogen mustard; releases ethyleneimine radicals that cross-link DNA. Crosses the blood-brain barrier, which underlies its use in CNS lymphoma conditioning.
- Approvals: US 1959: Breast and ovarian adenocarcinoma; superficial papillary bladder carcinoma (intravesical); malignant effusions; US 2017: Conditioning before allogeneic HSCT for class 3 beta-thalassaemia in children (Tepadina); EU 2010: Conditioning before haematopoietic progenitor cell transplantation for haematological diseases and solid tumours (Tepadina)

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Thiotepa
- US label (DailyMed): https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=thiotepa
- NCI drug page: https://www.cancer.gov/about-cancer/treatment/drugs/thiotepa
- EPAR (Tepadina): https://www.ema.europa.eu/en/medicines/human/EPAR/tepadina

## Connected records

- cancers: [Bladder & urothelial cancer](https://onco.cc/cancers/urothelial/), [Central nervous system germ cell tumours (germinoma and non-germinomatous)](https://onco.cc/cancers/cns-germ-cell-tumours/), [Diffuse large B-cell lymphoma](https://onco.cc/cancers/dlbcl/), [Group 3 and group 4 medulloblastoma (non-WNT/non-SHH)](https://onco.cc/cancers/medulloblastoma-group-3-4/), [High-risk neuroblastoma](https://onco.cc/cancers/neuroblastoma-high-risk/), [HR-positive / HER2-negative breast cancer](https://onco.cc/cancers/breast-hr-positive/), [Ovarian cancer](https://onco.cc/cancers/ovarian/), [Primary CNS lymphoma](https://onco.cc/cancers/primary-cns-lymphoma/), [SHH-activated medulloblastoma](https://onco.cc/cancers/medulloblastoma-shh/)
- technologies: [Allogeneic stem cell transplantation](https://onco.cc/technologies/allogeneic-hsct/), [Autologous stem cell transplant (high-dose therapy)](https://onco.cc/technologies/autologous-stem-cell-transplant/), [Cytotoxic chemotherapy](https://onco.cc/technologies/cytotoxic-chemotherapy/), [Intravesical therapy (BCG, chemotherapy, devices, gene and viral therapy)](https://onco.cc/technologies/bcg-and-intravesical-therapy/)
- companies: [Adienne Pharma & Biotech](https://onco.cc/companies/adienne/)
- trials: [ACNS0122](https://onco.cc/trials/acns0122/), [ACNS0333](https://onco.cc/trials/acns0333/), [ALL-SCT BFM International- HSCT in Children and Adolescents With ALL](https://onco.cc/trials/nct01423500/), [High-Risk Neuroblastoma Study 2 of SIOP-Europa-Neuroblastoma (SIOPEN)](https://onco.cc/trials/nct04221035/), [IELSG32](https://onco.cc/trials/ielsg32/), [MATRix/IELSG43](https://onco.cc/trials/ielsg43/), [Neo Adjuvant Chemotherapy in Triple Negative Breast Cancer](https://onco.cc/trials/nct01057069/)
- terms: [Stem cell transplant in lymphoma: what it is still for](https://onco.cc/terms/lymphoma-tx-transplant-role/), [The lymphoma regimen alphabet: R-CHOP, pola-R-CHP, DA-EPOCH-R, ABVD, BEACOPP and the rest](https://onco.cc/terms/lymphoma-tx-regimen-alphabet/)
- key papers: [IELSG32 first randomisation: the MATRix regimen (methotrexate, cytarabine, thiotepa, rituximab) in primary CNS lymphoma](https://onco.cc/key-papers/paper-ielsg32-matrix-induction-ferreri-lancet-haematol-2016/), [IELSG32 second randomisation: whole-brain radiotherapy or autologous stem cell transplant as consolidation in primary CNS lymphoma](https://onco.cc/key-papers/paper-ielsg32-wbrt-vs-asct-consolidation-ferreri-lancet-haematol-2017/), [MATRix/IELSG43: high-dose chemotherapy and autologous stem cell transplant versus non-myeloablative consolidation in primary CNS lymphoma](https://onco.cc/key-papers/paper-matrix-ielsg43-asct-consolidation-pcnsl-illerhaus-lancet-2026/)

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