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    DRUG:

    melphalan

    i
    Other names: L-PAM, L-sarcolysin, phenylalanine mustard
    Company:
    Generic mfg.
    Drug class:
    Alkylating agent
    Related drugs:
    1d
    Real MM: STUDY COMPARING TWO STANDARD TREATMENTS IN AUTOLOGOUS STEM CELL TRANSPLANTATION INELIGIBLE POPULATION AFFECTED BY MULTIPLE MYELOMA (clinicaltrials.gov)
    P4, N=450, Active, not recruiting, University of Turin, Italy | Recruiting --> Active, not recruiting
    Enrollment closed
    |
    lenalidomide • bortezomib • prednisone • melphalan • Darzalex Faspro (daratumumab and hyaluronidase-fihj) • dexamethasone injection
    1d
    Model-based antithymocyte globulin dosing in ex vivo CD34+ selected allogeneic haematopoietic cell transplantation: a single-centre, single-arm, phase 2 study. (PubMed, Lancet Haematol)
    These results demonstrate that model-based ATG dosing promotes robust CD4+ immune reconstitution after ex vivo CD34+ selected allogeneic HCT, underscoring the potential of pharmacokinetically guided ATG as a strategy to optimise immune recovery in myeloablative, calcineurin inhibitor-free transplantation for haematological malignancies.
    P2 data • Preclinical • Journal
    |
    CD34 (CD34 molecule)
    |
    cyclophosphamide • melphalan • fludarabine IV • thiotepa • busulfan
    2d
    Targeting MCM6 Enhances Melphalan Chemosensitivity in Retinoblastoma by Modulating DNA Damage Response. (PubMed, Invest Ophthalmol Vis Sci)
    MCM6 acts as a critical regulator of retinoblastoma growth and modulates response to melphalan. Targeting MCM6 may offer a therapeutic approach to improve outcomes of chemotherapy in retinoblastoma.
    Journal
    |
    MCM6 (Minichromosome Maintenance Complex Component 6)
    |
    melphalan
    2d
    Modified Conditioning Regimen for CML-BP (clinicaltrials.gov)
    P2, N=40, Not yet recruiting, Institute of Hematology & Blood Diseases Hospital, China
    New P2 trial
    |
    melphalan • fludarabine IV • thiotepa
    3d
    CD45RA-depleted CD19-CAR T Cell Consolidation After TCR??+ T Cell-depleted Haploidentical Hematopoietic Cell Transplantation for Relapsed/Refractory CD19+ ALL and Lymphoma (clinicaltrials.gov)
    P1, N=60, Not yet recruiting, St. Jude Children's Research Hospital
    New P1 trial
    |
    cyclophosphamide • melphalan • fludarabine IV • thiotepa • Neupogen (filgrastim)
    9d
    Study of the Impact of CD34+ Cell Dose on Absolute Lymphocyte Count Following High-Dose Therapy and Autologous Stem Cell Transplantation for Relapsed and Refractory Diffuse Large B-cell Lymphoma (DLBCL) (clinicaltrials.gov)
    P2, N=59, Active, not recruiting, Memorial Sloan Kettering Cancer Center | Trial completion date: Oct 2025 --> Oct 2026 | Trial primary completion date: Oct 2025 --> Oct 2026
    Trial completion date • Trial primary completion date
    |
    cytarabine • etoposide IV • carmustine • melphalan • plerixafor
    16d
    GEM-BELA-VRd: Belantamab Mafodotin in Newly Diagnosed Transplant Eligible Multiple Myeloma Patients (clinicaltrials.gov)
    P2, N=50, Active, not recruiting, PETHEMA Foundation | Recruiting --> Active, not recruiting | Trial completion date: Jul 2025 --> Dec 2025
    Enrollment closed • Trial completion date
    |
    lenalidomide • bortezomib • melphalan • Blenrep (belantamab mafodotin-blmf)
    16d
    NGS-based monitoring of secondary acute lymphoblastic leukemia dynamics following lenalidomide maintenance in multiple myeloma patients. (PubMed, Leuk Lymphoma)
    We report on a series of three patients diagnosed with secondary acute lymphoblastic leukemia (sALL) following treatment for multiple myeloma (MM) where serial, commercially available, next-generation sequencing (NGS) based tracking of measurable residual disease (MRD) using the clonoSEQ assay provided valuable clinical insight. Each of the patients in this series had been treated for their MM with regimens that had included autologous stem cell transplantation following high dose melphalan chemotherapy and had been on maintenance treatment with the immunomodulatory drug lenalidomide for at least one year.
    Journal • Next-generation sequencing
    |
    clonoSEQ
    |
    lenalidomide • melphalan
    17d
    Trial Evaluating MGTA-456 in Patients With High-Risk Malignancy (clinicaltrials.gov)
    P2, N=22, Active, not recruiting, Masonic Cancer Center, University of Minnesota | Completed --> Active, not recruiting | Trial completion date: Dec 2023 --> Dec 2026
    Enrollment closed • Trial completion date
    |
    FLT3 (Fms-related tyrosine kinase 3) • NPM1 (Nucleophosmin 1) • KMT2A (Lysine Methyltransferase 2A) • HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • CEBPA (CCAAT Enhancer Binding Protein Alpha)
    |
    FLT3-ITD mutation • NPM1 mutation • MLL rearrangement
    |
    cyclophosphamide • melphalan • fludarabine IV • busulfan • spanlecortemlocel (MGTA-456)
    21d
    Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia (clinicaltrials.gov)
    P2, N=72, Active, not recruiting, City of Hope Medical Center | Trial completion date: Oct 2025 --> Sep 2026
    Trial completion date
    |
    HLA-B (Major Histocompatibility Complex, Class I, B) • HLA-C (Major Histocompatibility Complex, Class I, C)
    |
    sirolimus • clofarabine • melphalan
    21d
    Ruxolitinib Before, During and After Hematopoietic Cell Transplant in Older Patients With Myelofibrosis and Myelodysplastic Syndrome/Myeloproliferative Neoplasm Overlap Syndromes (clinicaltrials.gov)
    P2, N=50, Not yet recruiting, Fred Hutchinson Cancer Center
    New P2 trial
    |
    Jakafi (ruxolitinib) • cyclophosphamide • methotrexate • melphalan • fludarabine IV • busulfan • methotrexate IV
    22d
    Allogeneic transplantation in adverse-risk acute myeloid leukemia: Challenges, strategies, and future directions. (PubMed, Chin J Cancer Res)
    Allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the cornerstone of consolidation for eligible patients; however, due to high relapse rates, regimen-related toxicity, and variable responses across genetic subtypes, optimal transplant timing, conditioning regimens (e.g., busulfan- or melphalan-based protocols), and bridging strategies require further refinement. Meanwhile, post-transplant maintenance therapies, such as hypomethylating agents or targeted drugs, are one emerging area under investigation for relapse prevention. In this perspective, we review the latest advances in allo-HSCT strategies for adverse-risk AML and highlight the importance of molecularly-guided approaches to improve outcomes in these aggressive subtypes.
    Journal
    |
    TP53 (Tumor protein P53) • NUP98 (Nucleoporin 98 And 96 Precursor 2) • NUP214 (Nucleoporin 214) • FUS (FUS RNA Binding Protein)
    |
    TP53 mutation
    |
    melphalan • busulfan