Muphoran (fotemustine) / Servier - LARVOL DELTA

Correlation of ten-year survival with cell-free DNA methylation of melanoma patients with asymptomatic brain metastases treated with nivolumab plus ipilimumab: The multicenter phase III NIBIT-M2 trial. (ASCO 2026) - P3 | " The NIBIT-M2 study recruited melanoma pts with active, untreated, asymptomatic BM from 9 Italian Centers, randomized (1:1:1) to receive fotemustine (F) (Arm A), I+F (Arm B), or I+N (Arm C). The 10-y results of the NIBIT-M2 study, with the longest follow-up available to date in melanoma pts with asymptomatic BM treated with I+N, continue to show persistent long-term therapeutic efficacy of the combination. Plasma-derived TF and T-meth Score may predict long-term survival of melanoma pts with asymptomatic BM treated with I+N."

cfDNA • Clinical • Epigenetic controller • P3 data • Melanoma • Solid Tumor

AMPK Pathway Activation Markers in GBM: Expression Patterns and Clinical Associations in a Real-World Study. (PubMed, Int J Mol Sci) - "This study aimed to characterize pACC and phosphorylated AMPK (pAMPK) expression in a real-world GBM cohort and to assess their prognostic and predictive value in patients receiving second-line regorafenib or fotemustine/lomustine. Although pACC-positive expression was enriched among patients experiencing longer overall survival under regorafenib, formal treatment-by-biomarker interaction was non-significant. These exploratory findings indicate that pACC is not an established predictive biomarker and warrant prospectively powered evaluation."

Biomarker • Journal • Real-world evidence • Retrospective data • Brain Cancer • Glioblastoma • Oncology • Solid Tumor • STK11

Ten-year survival and cell-free DNA methylation profiling of melanoma patients with asymptomatic brain metastases treated with nivolumab plus ipilimumab: the multicenter phase III NIBIT-M2 trial (EANO 2026) - "Here, we report the final analysis of the NIBIT-M2 study with the 10-y survival and its correlation with cell free (cf) DNA profiles.Material and The NIBIT-M2 study recruited melanoma pts with active, untreated, asymptomatic BM from 9 Italian Centers, randomized (1:1:1) to receive fotemustine (F) (Arm A), I+F (Arm B), or I+N (Arm C). The 10-y results of the NIBIT-M2 study, with the longest follow-up available to date in melanoma pts with asymptomatic BM, continue to show persistent long-term therapeutic efficacy of I+N. Plasma-derived TF and T-meth Score may predict long-term survival of melanoma pts with asymptomatic BM treated with I+N."

cfDNA • Clinical • Epigenetic controller • P3 data • Melanoma • Oncology • Solid Tumor

Determinants of clinical decision-making in first-line treatment of pleomorphic xanthoastrocytoma and the impact of BRAF status on subsequent therapies: a 10-year single-center series (EANO 2026) - "Grade 3 PXA pts underwent multimodality treatment, with 90% (9/10) receiving either radiochemotherapy with concurrent and adjuvant temozolomide (TMZ) (n=6) or radiotherapy followed by TMZ (n=3)...Second-line systemic chemotherapies included TMZ (n=4, mPFS 3.4 months) and fotemustine/lomustine with or without procarbazine (n=3, mPFS 4.5 m). Targeted therapies administered at first or second progression included dabrafenib/trametinib (n=2, DCR 50%, mPFS 25 m), vemurafenib (n=1, DCR 100%, mPFS 27 m), and regorafenib (n=1, DCR 0%, mPFS 1.1 m)... First-line treatment for PXA is primarily driven by grade, extent of resection, and patient-related factors. Despite the high prevalence of BRAF V600E mutations, targeted therapy was utilized in a minority of cases, likely due to the long analysis period and evolving therapeutic standards, including the approval of dabrafenib/trametinib in Italy in 2024 for high-grade gliomas. The clinical impact of BRAF inhibition and its potential..."

Clinical • Astrocytoma • Brain Cancer • High Grade Glioma • Oncology • Pleomorphic Xanthoastrocytoma • BRAF

Real-world effectiveness of chemotherapy regimens after immunotherapy in patients with advanced melanoma in Spain: Results from the GEM1801 study. (PubMed, Cancer) - "As in previous studies, patients treated with chemotherapy after immunotherapy failure had poor outcomes, similar to those from pre-immunotherapy era, and should be used when no other options are available."

Journal • Real-world evidence • Melanoma • Oncology • Solid Tumor

AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION COMBINED WITH CD19 CAR-T CELL THERAPY FOR CENTRAL NERVOUS SYSTEM LYMPHOMA: A CLINICAL EFFICACY AND SAFETY ANALYSIS (EHA 2026) - "All patients successfully received bridging therapy consisting of fotemustine, pemetrexed, orelabrutinib, and dexamethasone, followed by conditioning regimens, which were BEAM (fotemustine, etoposide, cytarabine, and melphalan) in 2 patients and fotemustine, thiotepa, and teniposide in 5 patients. Two patients developed infections, both of which were controllable. . Summary/Conclusion ASCT combined with CD19 CAR-T therapy demonstrates excellent efficacy and a favorable safety profile with newly diagnosed and relapsed/refractory CNSL, warranting further validation in larger cohorts with extended follow-up."

CAR T-Cell Therapy • Clinical • B Cell Lymphoma • Bone Marrow Transplantation • CNS Lymphoma • Hematological Malignancies • Infectious Disease • Lymphoma • Non-Hodgkin’s Lymphoma • Primary Central Nervous System Lymphoma • Secondary Central Nervous System Lymphoma • Transplantation

Ten-year survival and cell-free DNA methylation profiling of melanoma patients with asymptomatic brain metastases treated with nivolumab plus ipilimumab: The multicenter phase III NIBIT-M2 trial (AACR 2026) - "Here, we report the final analysis of the NIBIT-M2 study with the 10-y survival and its correlation with cell free (cf) DNA profiles. The NIBIT-M2 study recruited melanoma pts with active, untreated, asymptomatic BM from 9 Italian Centers, randomized (1:1:1) to receive fotemustine (F) (Arm A), I+F (Arm B), or I+N (Arm C). The 10-y results of the NIBIT-M2 study, with the longest follow-up available to date in melanoma pts with asymptomatic BM, continue to show persistent long-term therapeutic efficacy of I+N. Plasma-derived TF and T-meth Score may predict long-term survival of melanoma pts with asymptomatic BM treated with I+N."

cfDNA • Clinical • Epigenetic controller • P3 data • Melanoma • Oncology • Solid Tumor

Extracranial Metastases in Glioblastoma, IDH-Wildtype: A Case Series. (PubMed, Diagnostics (Basel)) - "All patients underwent surgical resection followed by temozolomide-based chemoradiation; two received fotemustine and bevacizumab, and one underwent re-irradiation. EM can arise despite MGMT-promoter methylation in IDH-wildtype GBM. Imaging red flags (high perfusion, surface contact, necrotic/FDG-avid cervical nodes) and clinical cues (axial pain, cytopenias, neck masses) should prompt early systemic staging (CT/PET-CT) and targeted tissue confirmation to advance management."

Journal • Back Pain • Brain Cancer • Glioblastoma • Hematological Disorders • Musculoskeletal Pain • Oncology • Pain • Solid Tumor • ATRX • GFAP • MGMT • OLIG2 • TERT • TP53

Fotemustine-containing chemotherapy suggests lower toxicity with comparable efficacy versus high-dose methotrexate-based regimens: a retrospective cohort analysis. (PubMed, Ther Adv Med Oncol) - "Notably, fotemustine-based therapy was associated with significantly fewer AEs, including leukopenia, thrombocytopenia, digestive tract toxicity, and mucositis (all p < 0.05). Fotemustine-containing chemotherapeutics appear to confer a safer profile with comparable efficacy relative to HD-MTX-based regimens in newly-diagnosed PCNSL patients."

Journal • Retrospective data • CNS Lymphoma • Hematological Disorders • Hematological Malignancies • Leukopenia • Lymphoma • Mucositis • Non-Hodgkin’s Lymphoma • Oncology • Primary Central Nervous System Lymphoma • Thrombocytopenia

Targeted and systemic therapies for recurrent adult ependymomas: real-world outcomes from a single institution and concise literature review. (PubMed, Front Oncol) - "Targeted therapy was administered to 7 patients: the TMZ-Lapatinib combination provided limited benefit (DCR 33%, median PFS 2.9 months), bevacizumab-based regimens showed variable efficacy; bevacizumab alone achieved a DCR of 33% with one case of prolonged stabilization (>58 months), while bevacizumab plus fotemustine yielded a PFS of 14.1 months. This real-world analysis indicates that temozolomide is feasible and associated with disease stabilization in selected patients, while bevacizumab-based combinations showed signals of clinical activity in recurrent adult ependymomas. Prospective, biomarker-driven multicenter trials are warranted to optimize systemic strategies in this rare disease."

Journal • Real-world evidence • Ependymoma • Oncology • Rare Diseases • Solid Tumor

A first-line regimen combining Bruton's tyrosine kinase and programmed cell death protein-1 inhibitors with chemotherapy excluding methotrexate achieves high response rates in primary central nervous system lymphoma. (PubMed, Cancer) - P1/2 | "Orelabrutinib combined with PD-1 blockade and fotemustine demonstrated high antitumor activity with manageable toxicity, supporting its potential as a frontline regimen for PCNSL."

Journal • B Cell Lymphoma • CNS Lymphoma • Hematological Disorders • Hematological Malignancies • Infectious Disease • Large B Cell Lymphoma • Leukopenia • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Primary Central Nervous System Lymphoma • Respiratory Diseases • Thrombocytopenia

Orelabrutinib plus anti-PD-1 antibody and fotemustine for newly diagnosed primary central nervous system lymphoma: Phase I/II results (ASH 2025) - P1/2 | "Orelabrutinib plus anti–PD-1 antibody and fotemustine demonstrated promising activity andacceptable safety in newly diagnosed PCNSL, with a high objective response rate. These findings supportthe potential of orelabrutinib-based chemoimmunotherapy as a promising frontline strategy. The studyis still ongoing and final results will be presented in the future."

P1/2 data • B Cell Lymphoma • CNS Lymphoma • Diffuse Large B Cell Lymphoma • Infectious Disease • Leukopenia • Lymphoma • Neutropenia • Non-Hodgkin’s Lymphoma • Primary Central Nervous System Lymphoma • Respiratory Diseases • Thrombocytopenia

Strikingly Reduced Toxicities of Fotemustine-Based Chemotherapeutics Than High-Dose Methotrexate-Containing Regimens with Comparable Efficacy (ASH 2024) - "Therefore, this study increased the sample size, extended the follow-up time and set up a control group to analyze the efficacy and safety of fotemustine-containing regimens compared with HD-MTX-containing regimens in the treatment of newly diagnosed PCNSL patients.Methods : From April 2011 to December 2021, 114 patients with newly diagnosed PCNSL who received HD-MTX-containing regimens (HD-MTX plus cytarabine [HD-MA], rituximab, HD-MTX plus temozolomide [R-MT]) or fotemustine-containing regimens (fotemustine, teniposide plus dexamethasone [FTD], fotemustine, temozolomide plus dexamethasone [FVD], rituximab, fotemustine, pemetrexed plus dexamethasone [RFPD]) were retrospectively analyzed in this study...Neither the progression free survival (PFS) (P=0.783) nor the overall survival (OS) (P=0.918) exhibited remarkably difference between HD-MTX-containing group and fotemustine-containing group. Notably, we noted that patients treated with HD-MTX-containing regimens..."

Clinical • Anemia • Brain Cancer • CNS Lymphoma • Hematological Disorders • Hematological Malignancies • Leukopenia • Lymphoma • Melanoma • Non-Hodgkin’s Lymphoma • Oncology • Primary Central Nervous System Lymphoma • Solid Tumor • Thrombocytopenia

SUCCESSFUL FIRST-LINE TREATMENT OF ADVANCED-STAGE HEPATIC HISTIOCYTIC SARCOMA WITH TANDEM AUTOLOGOUS AND ALLOGENEIC STEM CELL TRANSPLANTATION: A CASE REPORT (SIE 2025) - "Given the patient's fitness and the aggressive course of the disease, chemotherapy was escalated to ICE regimen (etoposide, carboplatin, ifosfamide) for four cycles. Following complete metabolic response on PET, the patient underwent ASCT after FEAM (fotemustine, etoposide, cytarabine, melphalan) conditioning, without any complication...The conditioning regimen consisted of thiotepa and melphalan. GVHD prophylaxis included post-transplant cyclophosphamide, tacrolimus, and mycophenolate mofetil...Systemic HS remains a clinical challenge due to its poor prognosis. This case report may support the hypothesis that upfront high-dose chemotherapy combined with allogeneic cellular therapy could play a potential role in achieving durable complete remission."

Case report • Clinical • Metastases • Cholestasis • Graft versus Host Disease • Hematological Malignancies • Hepatology • Immunology • Lymphoma • Sarcoma • Solid Tumor • Transplantation • BRAF • CD163 • CD4 • CD68 • KRAS • MAP2K1 • NRAS

STRIKINGLY REDUCED TOXICITIES OF FOTEMUSTINE-BASED CHEMOTHERAPEUTICS THAN HIGH-DOSE METHOTREXATE-CONTAINING REGIMENS WITH COMPARABLE EFFICACY (ICML 2025) - " From April 2011 to December 2021, 114 patients with newly diagnosed PCNSL who received HD-MTX-containing regimens (HD-MTX plus cytarabine [HD-MA], rituximab, HD-MTX plus temozolomide [R-MT]) or fotemustine-containing regimens (fotemustine, teniposide plus dexamethasone [FTD], fotemustine, temozolomide plus dexamethasone [FVD], rituximab, fotemustine, pemetrexed plus dexamethasone [RFPD]) were retrospectively analyzed in this study. Fotemustine-based chemotherapeutics conferred a safer effect on newly-diagnosed PCNSL patients compared with HD-MTX-containing regimens together with comparable efficiency."

Clinical • CNS Lymphoma • Lymphoma • Non-Hodgkin’s Lymphoma • Primary Central Nervous System Lymphoma

A PHASE I/II STUDY OF ORELABRUTINIB COMBINED WITH ANTI-PD-1 ANTIBODY AND FOTEMUSTINE FOR PATIENTS WITH NEWLY DIAGNOSED PRIMARY CENTRAL NERVOUS SYSTEM LYMPHOMA (ICML 2025) - P1/2 | "The combination of orelabrutinib, anti-PD-1 antibody, and fotemustine showed therapeutic potential in newly diagnosed PCNSL pts. However, clinically significant adverse events, particularly grade 3/4 thrombocytopenia and pulmonary infections, necessitate vigilant monitoring and proactive management to optimize patient outcomes."

Clinical • P1/2 data • CNS Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Primary Central Nervous System Lymphoma

A PHASE I/II STUDY OF ORELABRUTINIB COMBINED WITH ANTI-PROGRAMMED CELL DEATH PROTEIN-1 ANTIBODY AND FOTEMUSTINE FOR PATIENTS WITH NEWLY DIAGNOSED PRIMARY CENTRAL NERVOUS SYSTEM LYMPHOMA (PCNSL) (EHA 2025) - P1/2 | "The combination of orelabrutinib, anti-PD-1 antibody, and fotemustine showed therapeutic potential in newly diagnosed PCNSL pts. However, clinically significant AEs, particularly grade 3/4 thrombocytopenia and pulmonary infections, necessitate vigilant monitoring and proactive management to optimize patient outcomes."

Clinical • P1/2 data • Anemia • CNS Lymphoma • Dermatology • Diffuse Large B Cell Lymphoma • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Leukopenia • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Primary Central Nervous System Lymphoma • Psoriasis • Respiratory Diseases • Thrombocytopenia

Quantifying Nitrosoureas: Comprehensive Insights into Analytical Techniques Transforming Cancer Research. (PubMed, Crit Rev Anal Chem) - "This review provides a comprehensive overview of the analytical methodologies employed over the past forty years for the detection and quantification of five widely used nitrosourea-based anticancer drugs-carmustine (BCNU), lomustine (CCNU), semustine (methyl-CCNU), nimustine (NMT), and fotemustine (FM)-along with their metabolites. Furthermore, advancements in hybrid analytical techniques and their potential in enhancing the selectivity and sensitivity of NU detection are explored. This review aims to provide insights into the evolution of analytical approaches for nitrosourea-based drugs, highlighting their applications in pharmacokinetics, therapeutic drug monitoring, and environmental surveillance."

Journal • Review • Brain Cancer • Hematological Malignancies • Immunology • Lymphoma • Oncology • Pulmonary Disease • Respiratory Diseases • Solid Tumor

BEAM VS. EAM, FEAM, BEEAM, AND TEAM: EXPLORING CONDITIONING STRATEGIES IN AUTOLOGOUS STEM CELL TRANSPLANTATION FOR HODGKIN LYMPHOMA A RETROSPECTIVE STUDY OF LWP (EBMT 2025) - "BEAM (carmustine, etoposide, cytarabine, and melphalan) remains the most widely used conditioning regimen for lymphomas in Europe. However, alternatives have been developed over time to address challenges such as limited availability, high costs, or potential toxicities, notably including BeAM (substituting carmustine with bendamustine), FEAM (using fotemustine instead of carmustine), and TEAM (incorporating thiotepa)... We observed poorer outcomes for patients receiving BeAM, and better in those treated with FEAM compared to BEAM, while other conditioning regimens yielded comparable results."

Retrospective data • Bone Marrow Transplantation • Hematological Malignancies • Hodgkin Lymphoma • Lymphoma • Oncology • Transplantation

AUTOLOGOUS HSCT ACTIVITIES IN THE ERA OF IEC THERAPY: REPORT FROM A SINGLE CENTER (EBMT 2025) - "Background: Autologous HSCT in the era of Rituximab still represents a therapeutic option for patients with high risk DLBCL...The main conditioning regimen was the association of busulfan and melphalan until 2012 then we shifted to the use of the association of cytarabine, fotemustine, etoposide and melphalan... The introduction of IEC therapy represents a revolution as therapeutic alternative for high risk lymphoma, with probable and possible changes in the therapeutic algorithm. Despite that the use of high dose chemotherapy treatment with autologous HSCT consolidation, remain a standard of care stable over the years."

Clinical • Bone Marrow Transplantation • Diffuse Large B Cell Lymphoma • Hematological Disorders • Hematological Malignancies • Infectious Disease • Lymphoma • Oncology • Septic Shock

Fotemustine in Treating Patients With Metastatic Melanoma (clinicaltrials.gov) - P2 | N=12 | Terminated | Sponsor: Institut du Cancer de Montpellier - Val d'Aurelle | N=60 ➔ 12 | Completed ➔ Terminated; In view of the slowness of the inclusions and the difficulty of carrying out the study, it was decided to put an end to this study.

Enrollment change • Trial termination • Melanoma • Oncology • Solid Tumor • MGMT

A novel machine learning model integrating clinical and molecular data to predict response to second line treatment in recurrent IDHwtglioblastoma (AIOM 2024) - "Background : Nitrosoureas (lomustine/fotemustine) and antiangiogenic drugs (bevacizumab or regorafenib) are second-line treatment options for patients with recurrent IDHwt-glioblastoma (rGBM). The multi-classification ML model developed in this study was able to identify clinical and molecular signatures of recurrent glioblastoma patients responding to specific second-line treatment with bevacizumab or regorafenib or nitrosoureas."

Clinical • Machine learning • Brain Cancer • CNS Tumor • Glioblastoma • Oncology • Solid Tumor • CDKN2A • CDKN2B • EGFR • MTAP • PTEN • TP53

PTEN alteration as a predictor of second-line efficacy in patients with recurrent IDHwt-glioblastoma (rGBM) (AIOM 2024) - "Nitrosoureas (NS) such as lomustine and fotemustine and antiangiogenic drugs such as regorafenib(Reg) and bevacizumab (Bev) are all treatment options for rGBM. We concluded that pathogenic PTEN alteration may be a predictor of poor efficacy of regorafenib and lomustine in rGBM patients. However, a prospective study with a larger population is needed to better define the role of pTEN in these patient populations."

Clinical • Brain Cancer • CNS Tumor • Glioblastoma • Oncology • Solid Tumor • MGMT • PTEN

Fotemustine in recurrent high‑grade glioma: MRI neuro‑radiological findings. (PubMed, Oncol Lett) - "In conclusion, the present study described a series of patients with recurrent HGG treated with FTM in monotherapy, demonstrating a prevalent increase in lesion enhancement and three cases of marked restrictions on diffusion-weighted imaging. Further prospective studies are required to corroborate such preliminary results."

Journal • Astrocytoma • Brain Cancer • CNS Tumor • Glioblastoma • Glioma • Oncology • Solid Tumor

HOW I TREAT SARS-COV-2 INFECTION DURING HEMATOPOIETIC STEM CELL TRANSPLANT BONE MARROW APLASIA: MONOCENTRIC EXPERIENCE (SIE 2024) - "It was conditioned according to FEAM protocol (fotemustine, cytarabine, etoposide and mephalan) and was undergoing to infusion of CD34-positive cell dose 7.38 x106/kg, on day 0...The patient received antiviral therapy: remdesivir 200 mg on day -2 and 100 mg on day -1 and day 0. The patient has febrile neutropenia on day +7 and he received empirical antibiotic therapy with piperacillin tazobactam, the infectious agent could not be determined, and the fever disappeared within 24 hours...Conclusion. cases of SARS-CoV-2 infection in aplasia after high-dose regimen for autologous stem cell transplantation are rare in the medical literature; in our experience, the low viral load, the absence of comorbidities, but also the promptly CT scan and the start of antiviral therapy have allowed us to have no complications from SARS-CoV-2."

Aplastic Anemia • Bone Marrow Transplantation • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Hodgkin Lymphoma • Infectious Disease • Interstitial Lung Disease • Lymphoma • Neutropenia • Novel Coronavirus Disease • Oncology • Pneumonia • Respiratory Diseases • Transplantation