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    DRUG:

    Grafapex (treosulfan)

    i
    Other names: NSC 39069, CB 2562, CB 40067, CCRIS 2781, HSDB 6963
    Company:
    Medac, Medexus
    Drug class:
    Alkylating agent
    Related drugs:
    2d
    Effect of Moderate Renal Impairment and Race/Ethnicity on Treosulfan Pharmacokinetics (clinicaltrials.gov)
    P1, N=36, Recruiting, medac GmbH | Trial completion date: Dec 2025 --> Dec 2026 | Trial primary completion date: Aug 2025 --> Mar 2026
    Trial completion date • Trial primary completion date
    |
    HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1)
    |
    fludarabine IV • Grafapex (treosulfan)
    17d
    Safety and Efficacy of FT14 Conditioning for Allogeneic HSCT in Acute Myeloid Leukemia (clinicaltrials.gov)
    P2, N=82, Active, not recruiting, Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia
    New P2 trial
    |
    fludarabine IV • Grafapex (treosulfan)
    2ms
    Venetoclax- Augmented Treosulfan-Based Reduced Intensity Conditioning Before Allogeneic Stem Cell Transplantation (clinicaltrials.gov)
    P2, N=27, Not yet recruiting, University Hospital Tuebingen
    New P2 trial
    |
    Venclexta (venetoclax) • fludarabine IV • Grafapex (treosulfan)
    2ms
    Remission status prior allogeneic stem cell transplantation in AML/MDS patients harboring single-hit or multi-hit p53 mutations does not impact outcome. (PubMed, Transplant Cell Ther)
    The multi-hit TP53 mutation was found to be significantly associated with a reduced LFS rate in MDS/AML patients, in comparison to those with a single-hit TP53 mutation. Patients with a single or multi-hit TP53 may achieve a long-term survival rate of approximately 30%, and should be considered as transplant candidates, even in the absence of complete remission.
    Journal • P53mut
    |
    TP53 (Tumor protein P53)
    |
    TP53 mutation
    |
    fludarabine IV • Grafapex (treosulfan)
    3ms
    Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) (clinicaltrials.gov)
    P2, N=40, Active, not recruiting, Fred Hutchinson Cancer Center | Trial completion date: Dec 2026 --> Mar 2026
    Trial completion date
    |
    HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • HLA-DQB1 (Major Histocompatibility Complex, Class II, DQ Beta 1) • GATA2 (GATA Binding Protein 2) • HLA-B (Major Histocompatibility Complex, Class I, B) • HLA-C (Major Histocompatibility Complex, Class I, C)
    |
    LDH elevation
    |
    methotrexate • fludarabine IV • busulfan • Grafapex (treosulfan) • methotrexate IV
    3ms
    Treosulfan-Based Versus Clofarabine-Based Conditioning Before Donor Hematopoietic Stem Cell Transplant for the Treatment of Myelodysplastic Syndromes or Acute Myeloid Leukemia (clinicaltrials.gov)
    P2, N=23, Active, not recruiting, Fred Hutchinson Cancer Center | Recruiting --> Active, not recruiting | N=80 --> 23 | Trial completion date: Apr 2027 --> Jul 2026 | Trial primary completion date: Oct 2026 --> Jan 2026
    Enrollment closed • Enrollment change • Trial completion date • Trial primary completion date
    |
    HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • HLA-DQB1 (Major Histocompatibility Complex, Class II, DQ Beta 1) • HLA-B (Major Histocompatibility Complex, Class I, B) • HLA-C (Major Histocompatibility Complex, Class I, C)
    |
    clofarabine • fludarabine IV • busulfan • Grafapex (treosulfan)
    6ms
    Allogeneic haematopoietic stem cell transplantation for STAT3 hyper-IgE syndrome: a worldwide study. (PubMed, Blood Adv)
    Conditioning regimens were predominantly treosulfan-based (59%; with thiotepa, 34%); other patients received busulfan-based (24%) or melphalan-based (17%) regimens. This worldwide study expand data on HSCT for STAT3-HIES to 41 patients. Despite significant pre-HSCT pulmonary morbidity, OS was high, and patients have improved skin and respiratory disease, though the impact on extra-immune manifestations appears limited.
    Journal
    |
    STAT3 (Signal Transducer And Activator Of Transcription 3) • CD34 (CD34 molecule)
    |
    melphalan • thiotepa • busulfan • Grafapex (treosulfan)
    6ms
    RELEVANT: Randomized Evaluation of Treosulfan Versus Melphalan Conditioning Followed by PTCy in Patients With AML and MDS Undergoing Allogeneic Transplantation (clinicaltrials.gov)
    P2, N=220, Not yet recruiting, Technische Universität Dresden
    New P2 trial
    |
    melphalan • fludarabine IV • Grafapex (treosulfan)
    9ms
    Allogeneic Stem Cell Transplant for Myelofibrosis and Myelodysplastic Syndromes: A Contemporary Review. (PubMed, Am J Hematol)
    Harnessing molecular technologies, clinical expertise, patient-centered decision-making, and innovative pharmaceutical strategies offers an exciting opportunity to shape a transformative and curative treatment framework. Here, we provide a contemporary review on HCT for MDS and MF, highlighting up-to-date insights into disease biology, standard of care, and recommendations, as well as open avenues.
    Review • Journal
    |
    JAK2 (Janus kinase 2) • CALR (Calreticulin)
    |
    busulfan • Grafapex (treosulfan)
    9ms
    Haploidentical Hematopoietic Cell Transplantation in Dyskeratosis Congenita with Myelodysplastic Syndrome/Acute Myeloid Leukemia. (PubMed, Blood Cell Ther)
    He underwent allo-HCT with stable disease burden using a specifically-designed RIC regimen containing treosulfan (at 50% reduced dosing), fludarabine, and rabbit anti-thymocyte globulin. The graft versus host disease (GVHD) prophylaxis contained reduced-dose post-transplant cyclophosphamide (PTCy dose reduction of 50%) with mycophenolate mofetil and cyclosporine...This case demonstrates the safety and feasibility of haploidentical-HCT using a treosulfan-based reduced-intensity conditioning (RIC) regimen and modified PTCy-based GVHD prophylaxis in DKC. Disease relapse in this patient underscores the impact of pretransplant disease burden on relapse free survival in DKC patients with MDS/AML who are not eligible for myeloablative conditioning.
    Journal
    |
    RUNX1 (RUNX Family Transcription Factor 1) • DKC1 (Dyskerin Pseudouridine Synthase 1)
    |
    cyclophosphamide • fludarabine IV • Grafapex (treosulfan)
    9ms
    Treosulfan Therapeutic Drug Monitoring in Pediatric Hematopoietic Stem Cell Transplant Recipients (clinicaltrials.gov)
    P=N/A, N=70, Recruiting, Fondazione IRCCS Policlinico San Matteo di Pavia
    New trial
    |
    Grafapex (treosulfan)
    10ms
    Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) (clinicaltrials.gov)
    P2, N=40, Active, not recruiting, Fred Hutchinson Cancer Center | Recruiting --> Active, not recruiting
    Enrollment closed
    |
    HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • HLA-DQB1 (Major Histocompatibility Complex, Class II, DQ Beta 1) • GATA2 (GATA Binding Protein 2) • HLA-B (Major Histocompatibility Complex, Class I, B) • HLA-C (Major Histocompatibility Complex, Class I, C)
    |
    LDH elevation
    |
    methotrexate • fludarabine IV • busulfan • Grafapex (treosulfan) • methotrexate IV