Thiotepa

Mechanism-of-action class: Alkylating Activity.

• Thiotepa for Injection, USP has been tried with varying results in the palliation of a wide variety of neoplastic diseases. However, the most consistent results have been seen in the following tumors: 1.

• Thiotepa is contraindicated in patients with a known hypersensitivity (allergy) to this preparation. Therapy is probably contraindicated in cases of existing hepatic, renal, or bone-marrow damage.contraindicated

Since absorption from the gastrointestinal tract is variable, thiotepa should not be administered orally. Dosage must be carefully individualized. A slow response to thiotepa does not necessarily indicate a lack of effect. Therefore, increasing the frequency of dosing may only increase toxicity. After maximum benefit is obtained by initial therapy, it is necessary to continue the patient on maintenance therapy (1 to 4 week intervals). In order to continue optimal effect, maintenance doses should not be administered more frequently than weekly in order to preserve correlation between dose and blood counts. Preparation and Administration Precautions: Thiotepa is a cytotoxic anticancer drug and as with other potentially toxic compounds, caution should be exercised in handling and preparation of thiotepa. Skin reactions associated with accidental exposure to thiotepa may occur. The use of gloves is recommended. If thiotepa solution contacts the skin, immediately wash the skin thoroughly with soap and water. If thiotepa contacts mucous membranes, the membranes should be flushed thoroughly with water. Preparation of Solution: Thiotepa for injection should be reconstituted with 1.5 m L of sterile water for injection resulting in a drug concentration of approximately 10 mg/mL .

Death has occurred after intravesical administration, caused by bone-marrow depression from systematically absorbed drug. Death from septicemia and hemorrhage has occurred as a direct result of hematopoietic depression by thiotepa. Thiotepa is highly toxic to the hematopoietic system. A rapidly falling white blood cell or platelet count indicates the necessity for discontinuing or reducing the dosage of thiotepa. Weekly blood and platelet counts are recommended during therapy and for at least 3 weeks after therapy has been discontinued. Thiotepa can cause fetal harm when administered to a pregnant woman. Thiotepa given by the intraperitoneal (IP) route was teratogenic in mice at doses ≥ 1 mg/kg (3.2 mg/m 2 ), approximately 8-fold less than the maximum recommended human therapeutic dose (0.8 mg/kg, 27 mg/m 2 ), based on body-surface area. Thiotepa given by the IP route was teratogenic in rats at doses ≥ 3 mg/kg (21 mg/m 2 ), approximately equal to the maximum recommended human therapeutic dose, based on body-surface area. Thiotepa was lethal to rabbit fetuses at a dose of 3 mg/kg (41 mg/m 2 ), approximately two times the maximum recommended human therapeutic dose based on body-surface area. Effective contraception should be used during thiotepa therapy if either the patient or partner is of childbearing potential. There are no adequate and well-controlled studies in pregnant women. If thiotepa is used during pregnancy, or if pregnancy occurs during thiotepa therapy, the patient and partner should be apprised of the potential hazard to the fetus.

In addition to its effect on the blood-forming elements (see WARNINGS and PRECAUTIONS sections), thiotepa may cause other adverse reactions. General: Fatigue, weakness. Febrile reaction and discharge from a subcutaneous lesion may occur as the result of breakdown of tumor tissue. Hypersensitivity Reactions: Allergic reactions - rash, urticaria, laryngeal edema, asthma, anaphylactic shock, wheezing. Local Reactions: Contact dermatitis, pain at the injection site. Gastrointestinal: Nausea, vomiting, abdominal pain, anorexia. Renal: Dysuria, urinary retention. There have been rare reports of chemical cystitis or hemorrhagic cystitis following intravesical, but not parenteral administration of thiotepa. Respiratory: Prolonged apnea has been reported when succinylcholine was administered prior to surgery, following combined use of thiotepa and other anticancer agents. It was theorized that this was caused by decrease of pseudocholinesterase activity caused by the anticancer drugs. Neurologic: Dizziness, headache, blurred vision. Skin: Dermatitis, alopecia. Skin depigmentation has been reported following topical use. Special Senses: Conjunctivitis. Reproductive: Amenorrhea, interference with spermatogenesis.

Pregnancy Category D: See WARNINGS section. Thiotepa can cause fetal harm when administered to a pregnant woman. Thiotepa given by the IP route was teratogenic in mice at doses ≥ 1 mg/kg (3.2 mg/m 2 ), approximately 8-fold less than the maximum recommended human therapeutic dose based on body-surface area. Thiotepa given by the IP route was teratogenic in rats at doses ≥ 3 mg/kg (21 mg/m 2 ), approximately equal to the maximum recommended human therapeutic dose based on body-surface area. Thiotepa was lethal to rabbit fetuses at a dose of 3 mg/kg (41 mg/m 2 ), approximately 2 times the maximum recommended human therapeutic dose based on body-surface area. Patients of childbearing potential should be advised to avoid pregnancy. There are no adequate and well-controlled studies in pregnant women. If thiotepa is used during pregnancy, or if pregnancy occurs during thiotepa therapy, the patient and partner should be apprised of the potential hazard to the fetus.

Hematopoietic toxicity can occur following overdose, manifested by a decrease in the white cell count and/or platelets. Red blood cell count is a less accurate indicator of thiotepa toxicity. Bleeding manifestations may develop. The patient may become more vulnerable to infection, and less able to combat such infection. Dosages within and minimally above the recommended therapeutic doses have been associated with potentially life-threatening hematopoietic toxicity. Thiotepa has a toxic effect on the hematopoietic system that is dose related. Thiotepa is dialyzable. There is no known antidote for overdosage with thiotepa. Transfusions of whole blood or platelets have proven beneficial to the patient in combating hematopoietic toxicity.

• Apr 2, 2001ANDAANDA075547West-ward Pharms Int
• Jan 26, 2017NDANDA208264Adienne Sa
• May 4, 2018ANDAANDA210337Dr Reddys
• May 4, 2018ANDAANDA209150Hengrui Pharma
• Mar 4, 2020ANDAANDA213049Msn
• Mar 8, 2021ANDAANDA214222Gland
• Sep 5, 2023ANDAANDA211755Hikma
• Jun 25, 2024NDANDA216984Shorla

1. 1Off Label Use1,10912%
2. 2Febrile Neutropenia7968.6%
3. 3Mucosal Inflammation7938.6%
4. 4Product Use In Unapproved Indication5956.4%
5. 5Cytomegalovirus Infection5235.6%
6. 6Drug Ineffective5215.6%
7. 7Pyrexia5135.5%
8. 8Acute Graft Versus Host Disease4504.9%
9. 9Acute Graft Versus Host Disease In Skin4304.6%
10. 10Infection4184.5%
11. 11Neutropenia4074.4%
12. 12Cytomegalovirus Infection Reactivation3964.3%
13. 13Sepsis3944.3%
14. 14Venoocclusive Liver Disease3864.2%
15. 15Thrombocytopenia3824.1%

Recent PubMed references pinned to Thiotepa as a MeSH major topic. Citations link to pubmed.ncbi.nlm.nih.gov.

• Novel FABT-Based Conditioning Regimen With Haploidentical Transplantation for Severe Aplastic Anemia: A Prospective, Single-Center, Phase II Clinical Trial.
  Transplantation and cellular therapy · 2026 · Liu W, Tan Z, Zhao Y, et al.
  PMID 41730450DOI 10.1016/j.jtct.2025.12.942
• A retrospective analysis of autologous stem cell transplantation conditioning with reduced-dose busulfan/thiotepa for patients with central nervous system lymphomas at a single institution.
  International journal of hematology · 2026 · Hattori K, Kurita N, Matsumura F, et al.
  PMID 41400781DOI 10.1007/s12185-025-04130-w
• Alemtuzumab, Fludarabine, Melphalan, and Thiotepa Conditioning for Transplantation in Inborn Errors of Immunity.
  Transplantation and cellular therapy · 2026 · Pfeiffer T, Murray L, Gao F, et al.
  PMID 41274644DOI 10.1016/j.jtct.2025.11.028
• [The Efficacy and Safety of Modified Thiotepa-Based Conditioning Followed by Autologous Stem Cell Transplantation in Primary CNS Lymphomas].
  Zhongguo shi yan xue ye xue za zhi · 2025 · Li Y, Yang P, Bao F, et al.
  PMID 41234097DOI 10.19746/j.cnki.issn.1009-2137.2025.05.030
• Tailoring Intensity: A Perspective on Thiotepa, Busulfan, and Fludarabine (TBF) Conditioning for Acute Leukemias and Myeloproliferative Neoplasms.
  Transplantation and cellular therapy · 2026 · Abdel Rahman Z, Mansour R, Kharfan-Dabaja M, et al.
  PMID 41213501DOI 10.1016/j.jtct.2025.11.015
• [Thiotepa-containing conditioning for allogeneic hematopoietic stem cell transplantation in children with inborn errors of immunity: a retrospective clinical analysis].
  Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics · 2025 · Wu XJ, Han XW, Wang KM, et al.
  PMID 41121701DOI 10.7499/j.issn.1008-8830.2504147
• Thiotepa-busulfan-fludarabine compared to clofarabine-based conditioning for haploidentical transplant with posttransplant cyclophosphamide in patients with myeloid malignancies: a retrospective study from the SFGM-TC.
  Bone marrow transplantation · 2025 · Jullien M, Brissot E, Daguindau E, et al.
  PMID 40954245DOI 10.1038/s41409-025-02709-9
• Clinical Efficacy and Safety of a Modified Conditioning Regimen Reducing the Dosage of Busulfan and Adding Thiotepa in Allogeneic Hematopoietic Stem Cell Transplantation for Pediatric Acute Myeloid Leukemia.
  Transplantation proceedings · 2025 · Song N, Zheng M, Wu P, et al.
  PMID 40945969DOI 10.1016/j.transproceed.2025.07.027

The 10 most recently updated of 349 ClinicalTrials.gov registrations naming Thiotepa as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Dinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma
  Recruiting · Phase 3 · Interventional · 478 enrolled · National Cancer Institute (NCI)
  NCT06172296updated 2026-06-12
• Pilot Study of IT Topotecan and Maintenance Chemotherapy for HR-EBTs in Children < 6 Years, Post Consolidation
  Recruiting · Early phase 1 · Interventional · 15 enrolled · C17 Council
  NCT06942039updated 2026-06-08
• Allo HSCT for High Risk Hemoglobinopathies
  Recruiting · Phase 2 · Interventional · 62 enrolled · Masonic Cancer Center, University of Minnesota
  NCT06872333updated 2026-06-04
• Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease
  Recruiting · Phase 1 · Phase 2 · Interventional · 24 enrolled · Arkansas Children's Hospital Research Institute
  NCT07252050updated 2026-06-04
• HeadStart4: Newly Diagnosed Children (<10 y/o) With Medulloblastoma and Other CNS Embryonal Tumors
  Active not recruiting · Phase 4 · Interventional · 250 enrolled · Parth Patel
  NCT02875314updated 2026-06-03
• TCRαβ-depleted Progenitor Cell Graft With Additional Memory T-cell DLI, Plus Selected Use of Blinatumomab, in Naive T-cell Depleted Haploidentical Donor Hematopoietc Cell Transplantation for Hematologic Malignancies
  Active not recruiting · Phase 2 · Interventional · 69 enrolled · St. Jude Children's Research Hospital
  NCT03849651updated 2026-06-03
• Dinutuximab, Sargramostim, and Combination Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma
  Completed · Phase 2 · Interventional · 42 enrolled · National Cancer Institute (NCI)
  NCT03786783updated 2026-06-02
• A Multicenter, Prospective Clinical Trial With a Concurrent Control Evaluating Methotrexate Combined With Rituximab,Sintilimab and Pirtobrutinib vs. Investigator-Selected Standard of Care in Treatment-Naive PCNSL
  Recruiting · Phase 2 · Interventional · 77 enrolled · Tongji Hospital
  NCT07350850updated 2026-06-02
• TCRαβ-depleted Progenitor Cell Graft With Early Memory T-cell DLI, Plus Selected Use of Blinatumomab, in naïve T-cell Depleted Haploidentical Donor Hematopoietic Cell Transplantation for Hematologic Malignancies
  Recruiting · Phase 1 · Interventional · 30 enrolled · St. Jude Children's Research Hospital
  NCT07052370updated 2026-06-02
• Substantially Improving the Cure Rate of High-risk BRCA1-like Breast Cancer
  Active not recruiting · Phase 3 · Interventional · 174 enrolled · The Netherlands Cancer Institute
  NCT02810743updated 2026-06-01

Frequently asked questions

How does Thiotepa work?

Mechanism-of-action class: Alkylating Activity.

What is Thiotepa used for?

According to FDA labeling, Thiotepa carries indications including: Thiotepa for Injection, USP has been tried with varying results in the palliation of a wide variety of neoplastic diseases. However, the most consistent results have been seen in the following tumors: 1.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Thiotepa?

Thiotepa is classified as Ethylene imines, Alkylating Drug, Alkylating Activity, Decreased DNA Integrity, Decreased DNA Replication.

What are the brand names for Thiotepa?

Thiotepa is marketed under brand names including Tepadina, Tepylute.

What are the contraindications for Thiotepa?

Thiotepa labeling lists contraindications including: Thiotepa is contraindicated in patients with a known hypersensitivity (allergy) to this preparation. Therapy is probably contraindicated in cases of existing hepatic, renal, or bone-marrow damage.. Always consult the full prescribing information and a clinician.