Treosulfan

---

title: "Treosulfan" type: doc version: 1 created: 2026-02-28 author: "Wikipedia contributors" status: active scope: public tags: ["alkylsulfonates", "iarc-group-1-carcinogens", "alkylating-antineoplastic-agents", "mesylate-esters", "orphan-drugs", "vicinal-diols"] description: "Medication given to people before they have a bone marrow transplant" topic_path: "general/alkylsulfonates" source: "https://en.wikipedia.org/wiki/Treosulfan" license: "CC BY-SA 4.0" wikipedia_page_id: 0 wikipedia_revision_id: 0

::summary Medication given to people before they have a bone marrow transplant ::

::data[format=table title="Infobox drug"]

Field	Value
Verifiedfields	changed
Watchedfields	changed
verifiedrevid	447997363
image	Treosulfan.svg
image_class	skin-invert-image
tradename	Trecondi, others
Drugs.com
MedlinePlus	a625065
DailyMedID	Treosulfan
pregnancy_AU	D
pregnancy_AU_comment
routes_of_administration	By mouth, intravenous
ATC_prefix	L01
ATC_suffix	AB02
legal_AU	S4
legal_AU_comment
legal_BR
legal_CA	Rx-only
legal_CA_comment
legal_DE
legal_NZ
legal_UK	POM
legal_UK_comment
legal_US	Rx-only
legal_US_comment
legal_EU	Rx-only
legal_EU_comment
legal_UN
legal_status	Rx-only
CAS_number_Ref
CAS_number	299-75-2
PubChem	9882105
DrugBank_Ref
DrugBank	DB11678
ChemSpiderID	8057780
UNII_Ref
UNII	CO61ER3EPI
KEGG	C19557
KEGG2	D07253
ChEBI	82557
synonyms	1,2,3,4-Butanetetrol, 1,4-dimethanesulfonate, Threitol 1,4-dimethanesulfonate, Threitol 1,4-bismethanesulfonate; L-Threitol 1,4-bis(methanesulfonate); Threosulphan; Treosulphan; Tresulfan
IUPAC_name	(2S,3S)-2,3-Dihydroxybutane-1,4-diyl dimethanesulfonate
C	6
SMILES	CS(=O)(=O)OCC@@HO
StdInChI	1S/C6H14O8S2/c1-15(9,10)13-3-5(7)6(8)4-14-16(2,11)12/h5-8H,3-4H2,1-2H3/t5-,6-/m0/s1
StdInChIKey	YCPOZVAOBBQLRI-WDSKDSINSA-N
melting_point	101.5
melting_high	105
::

| Verifiedfields = changed | Watchedfields = changed | verifiedrevid = 447997363 | image = Treosulfan.svg | image_class = skin-invert-image | width = | alt = | caption =

| pronounce = | tradename = Trecondi, others | Drugs.com = | MedlinePlus = a625065 | DailyMedID = Treosulfan | pregnancy_AU = D | pregnancy_AU_comment = | pregnancy_category= | routes_of_administration = By mouth, intravenous | class = | ATC_prefix = L01 | ATC_suffix = AB02 | ATC_supplemental =

| legal_AU = S4 | legal_AU_comment = | legal_BR =
| legal_BR_comment = | legal_CA = Rx-only | legal_CA_comment = | legal_DE = | legal_DE_comment = | legal_NZ = | legal_NZ_comment = | legal_UK = POM | legal_UK_comment = | legal_US = Rx-only | legal_US_comment = | legal_EU = Rx-only | legal_EU_comment = | legal_UN = | legal_UN_comment = | legal_status = Rx-only

| bioavailability = | protein_bound = | metabolism = | metabolites = | onset = | elimination_half-life = | duration_of_action = | excretion =

| CAS_number_Ref = | CAS_number = 299-75-2 | CAS_supplemental = | PubChem = 9882105 | IUPHAR_ligand = | DrugBank_Ref = | DrugBank = DB11678 | ChemSpiderID_Ref = | ChemSpiderID = 8057780 | UNII_Ref = | UNII = CO61ER3EPI | KEGG_Ref = | KEGG = C19557 | KEGG2_Ref = | KEGG2 = D07253 | ChEBI_Ref = | ChEBI = 82557 | ChEMBL_Ref = | ChEMBL = | NIAID_ChemDB = | PDB_ligand = | synonyms = 1,2,3,4-Butanetetrol, 1,4-dimethanesulfonate, Threitol 1,4-dimethanesulfonate, Threitol 1,4-bismethanesulfonate; L-Threitol 1,4-bis(methanesulfonate); Threosulphan; Treosulphan; Tresulfan

| IUPAC_name = (2S,3S)-2,3-Dihydroxybutane-1,4-diyl dimethanesulfonate | C=6 | H=14 | O=8 | S=2 | SMILES = CS(=O)(=O)OCC@@HO | Jmol = | StdInChI = 1S/C6H14O8S2/c1-15(9,10)13-3-5(7)6(8)4-14-16(2,11)12/h5-8H,3-4H2,1-2H3/t5-,6-/m0/s1 | StdInChI_comment = | StdInChIKey = YCPOZVAOBBQLRI-WDSKDSINSA-N | density = | density_notes = | melting_point = 101.5 | melting_high = 105 | melting_notes = | boiling_point = | boiling_notes = | solubility = | sol_units = | specific_rotation =

Treosulfan, sold under the brand name Trecondi among others, is an alkylating medication given to people before they have a bone marrow transplant from a donor known as allogeneic hematopoietic stem cell transplantation. It is used as a 'conditioning' treatment to clear the bone marrow and make room for the transplanted bone marrow cells, which can then produce healthy blood cells. It is used together with another medicine called fludarabine in adults and children from one month of age with blood cancers as well as in adults with other severe disorders requiring a bone marrow transplant. It belongs to the family of drugs called alkylating agents. In the body, treosulfan is converted into other compounds called epoxides which kill cells, especially cells that develop rapidly such as bone marrow cells, by attaching to their DNA while they are dividing.

The most common side effects include infections, nausea (feeling sick), stomatitis (inflammation of the lining of the mouth), vomiting, diarrhea, and abdominal pain (belly ache). Tiredness, febrile neutropenia (low white blood cell counts with fever) and high blood levels of bilirubin (a breakdown product of red blood cells) are also seen in more than 1 in 10 adults, and rash also affects more than 1 in 10 children. The most common adverse reactions include musculoskeletal pain, stomatitis, pyrexia, nausea, edema, infection, and vomiting. Selected grade 3 or 4 nonhematological laboratory abnormalities include increased GGT, increased bilirubin, increased ALT, increased AST, and increased creatinine.

Treosulfan was authorized for medical use in the European Union in June 2019, and approved for medical use in the United States in January 2025.

Medical Uses

Treosulfan in combination with fludarabine is indicated as part of conditioning treatment prior to allogeneic haematopoietic stem cell transplantation in adults with malignant and non malignant diseases, and in children older than one month with malignant diseases.

History

Two main studies showed that treosulfan is at least as effective as busulfan, another medicine used to prepare people for haematopoietic stem cell transplantation.

In one of the studies, involving 570 adults with acute myeloid leukaemia (a blood cancer) or myelodysplastic syndromes (conditions in which large numbers of abnormal blood cells are produced), 64% of patients given treosulfan (with fludarabine) had a successful transplant and were alive and disease-free after 2 years, compared with 51% of patients given busulfan (with fludarabine).

In an additional study in 70 children with blood cancers, 99% of children given treosulfan (with fludarabine) were alive three months after their transplant.

Efficacy was evaluated in MC-FludT.14/L Trial II (NCT00822393), a randomized active-controlled trial comparing treosulfan to busulfan with fludarabine as a preparative regimen for allogeneic transplantation. Eligible patients included adults 18 to 70 years old with AML or MDS, Karnofsky performance status ≥ 60%, and age ≥ 50 years or hematopoietic cell transplantation comorbidity index [HCTCI] score 2. There were 570 patients randomized to treosulfan (n=280) or busulfan (n=290).

Society and culture

Legal status

Treosulfan was authorized for medical use in the European Union in June 2019, and approved for medical use in the United States in January 2025.

The US Food and Drug Administration granted orphan drug designation to treosulfan in 1994, for the treatment of ovarian cancer; and in 2015, for conditioning treatment prior to hematopoietic stem cell transplantation in malignant and non-malignant diseases in adults and pediatric patients.

In February 2004, orphan designation (EU/3/04/186) was granted by the European Commission to medac Gesellschaft fuer klinische Spezialpräparate mbH, Germany, for treosulfan for the conditioning treatment prior to haematopoietic progenitor cell transplantation.

Names

Treosulfan is the international nonproprietary name.

Treosulfan is sold under the brand names Trecondi, Grafapex and Ovastat.

References

References

1. (21 December 2022). "Updates to the Prescribing Medicines in Pregnancy database".
2. (11 October 2022). "Trecondi APMDS".
3. (14 January 2025). "Trecondi (Link Medical Products Pty Ltd T/A Link Pharmaceuticals)".
4. (4 July 2023). "AusPAR: Trecondi".
5. (3 August 2022). "Health product highlights 2021: Annexes of products approved in 2021".
6. "Treosulfan 5g Powder for Solution for Infusion - Summary of Product Characteristics (SmPC)".
7. (31 January 2025). "Grafapex- treosulfan injection, powder, lyophilized, for solution".
8. (21 April 2020). "Trecondi Product Information".
9. (11 December 2018). "Trecondi EPAR".
10. (October 2018). "Treosulfan Pharmacokinetics and its Variability in Pediatric and Adult Patients Undergoing Conditioning Prior to Hematopoietic Stem Cell Transplantation: Current State of the Art, In-Depth Analysis, and Perspectives". Clinical Pharmacokinetics.
11. (6 February 2025). "FDA approves treosulfan with fludarabine as a preparative regimen for alloHSCT in adult and pediatric patients with AML or MDS".
12. (21 February 2025). "Novel Drug Approvals for 2025".
13. (22 January 2025). "Medexus Announces FDA Approval of Grafapex (treosulfan) for Injection and Provides Business Update". Medexus Pharmaceuticals.
14. (16 May 1994). "Treosulfan Orphan Drug Designations and Approvals".
15. (8 April 2015). "Treosulfan Orphan Drug Designations and Approvals".
16. (17 September 2018). "EU/3/04/186".
17. (1972). "International nonproprietary names for pharmaceutical substances (INN). recommended INN: list 12". WHO Chronicle.
18. "A process for the preparation of treosulfan".
19. (25 November 2024). "Treosulfan (Code C1257)".

::callout[type=info title="Wikipedia Source"] This article was imported from Wikipedia and is available under the Creative Commons Attribution-ShareAlike 4.0 License. Content has been adapted to SurfDoc format. Original contributors can be found on the article history page. ::