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November 03, 2023
High-Dose Methotrexate Containing Induction Chemotherapy Followed By Nivolumab Consolidation in Older (≥ 65) Patients with Previously Untreated Primary CNS Lymphoma
(ASH 2023)
- P1 | "Variations of the following regimens were used for induction: 43% R-MPV (rituximab, methotrexate, procarbazine, and vincristine) (43%), 29% MRT (methotrexate, rituximab, and temozolomide), 21% MR (methotrexate and rituximab), and 7% MATRix (methotrexate, cytarabine, thiotepa, and rituximab)... The current study demonstrates encouraging safety and clinical outcomes of nivolumab consolidation in older PCNSL patients who are deemed poor candidates for ASCT or WBI. No DLT was observed during the safety run-in phase, and there was no unexpected toxicity associated with nivolumab although 1 patient discontinued the study treatment due to Stevens-Johnson syndrome, a rare but known AE associated with nivolumab, after Cycle 1. Overall, nivolumab consolidation was associated with favorable survival rates in a group of patients with poor-risk PCNSL."
Clinical • Acute Kidney Injury • CNS Lymphoma • Fatigue • Hematological Disorders • Hematological Malignancies • Infectious Disease • Lymphoma • Nephrology • Neutropenia • Non-Hodgkin’s Lymphoma • Oncology • Primary Central Nervous System Lymphoma • Renal Disease • Septic Shock • Steven-Johnson Syndrome
November 04, 2025
Myeloablative fractionated busulfan, fludarabine, cladribine, thiotepa, and venetoclax (Cladillac) conditioning for high-risk AML: A phase 2 trial
(ASH 2025)
- P2/3 | "To reduce GVHD-associated morbidity and mortality, weincorporated post-transplant cyclophosphamide (PTCy)...GVHD prophylaxis consisted of PTCy 50mg/kg on days +3 and +4, tacrolimus ± mycophenolate mofetil... This study met its primary endpoint, demonstrating a promising 3-year PFS of 58% in acohort of patients with very high-risk AML. Outcomes were particularly favorable in TP53 wild-typepatients, with a low relapse rate of 13% and 3-year OS of 74%. These findings support furtherinvestigation of this novel conditioning regimen."
P2 data • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • FLT3 • TP53
September 01, 2026
Peripheral T-Cell Lymphoma Not Otherwise Specified With Infratentorial Parenchymal Involvement in a Young Patient With Advanced Untreated HIV Infection: A Case Report
(SOHO 2026)
- "Given imaging characteristics and the epidemiology that ∼90% of primary CNS lymphomas are B-cell lymphomas, an urgent methotrexate, cytarabine, thiotepa, and rituximab (MATRix)-like induction regimen was started (December 6, 2025) to achieve intracranial control before nodal histopathology was available...After histologic confirmation, the treatment plan was revised to alternate brentuximab vedotin plus cyclophosphamide, doxorubicin, vincristine, and prednisone (BV-CHOP) with highdose methotrexate/cytarabine to address systemic CD30+ disease and maintain CNS control. This case illustrates (1) rationale for empiric MATRix initiation when clinical urgency and imaging strongly suggest primary B-cell CNS lymphoma, (2) the need to promptly adapt systemic therapy when final histology indicates a different lineage (PTCL NOS), and (3) the importance of multidisciplinary management imperatives in people living with HIV (PLWH) with aggressive lymphoma."
Case report • Clinical • Metastases • B Cell Lymphoma • CNS Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Peripheral T-cell Lymphoma • Primary Central Nervous System Lymphoma • T Cell Non-Hodgkin Lymphoma • CD20 • CD4 • CD7 • GATA3 • PAX5 • TNFRSF8
July 16, 2024
Intensified alkylating chemotherapy with autologous stem cell rescue (IACT) or conventional chemotherapy followed by olaparib (CCT-O) in stage III, HER2-negative, homologous recombination deficient (HRD) breast cancer (BC): Survival results of the randomized-controlled SUBITO trial
(ESMO 2024)
- P3 | "It is unknown whether adding olaparib to 3rd generation chemotherapy (long lasting regimen) can achieve similar OS as IACT (fast & forceful regimen) in pts with high-risk HRD BCs. In this multicenter, open-label phase III trial pts with stage III, HER2-negative, BRCA1-like or germline BRCA1/2 mutated (gBRCAm) BC were randomized 1:1 to IACT (4x dose-dense doxorubicin-cyclophosphamide (ddAC), followed by 2 cycles of carboplatin 800 mg/m2, thiotepa 250 mg/m2, and cyclophosphamide 3,000 mg/m2 with autologous stem cell rescue), or CCT-O (4xddAC-4x carboplatin-paclitaxel, and 1 year of olaparib). Adjuvant capecitabine was offered to pts without a pathological complete response (pCR) in both arms after an amendment in 2019... Both DNA double strand break-inducing regimens yield promising 4-year OS rates in pts with high-risk stage III, HER2-negative, HRD breast cancer, especially when reaching a pCR."
Clinical • Late-breaking abstract • Breast Cancer • HER2 Breast Cancer • HER2 Negative Breast Cancer • HER2 Positive Breast Cancer • Oncology • Solid Tumor • Triple Negative Breast Cancer • BRCA1 • BRCA2 • HER-2 • HRD
November 04, 2025
High event-free (EFS) and overall survival (OS) after non-total body irradiation (TBI) conditioning and allogeneic hematopoietic cell transplantation (HCT) in next-generation-sequencing minimal residual disease (NGS-MRD) negative B-acute lymphoblastic leukemia (B-ALL): Results from the EndRAD trial (PTCTC ONC1701)
(ASH 2025)
- P2 | "Based upon retrospectivedata showing low rates of relapse, we hypothesized that patients with negative pre-HCT MRD by next-generation-sequencing of IgH B-cell receptor rearrangements (NGS-MRD) could achieve 2-year EFSexceeding 75% with a non-TBI regimen, an outcome comparable to those receiving TBI-based regimens. The Pediatric Transplantation and Cellular Therapy Consortium (PTCTC) conducted a phase IIprospective trial at 45 Centers in North America (ONC1701 EndRAD: NCT03509961) between 2018 and2025 to evaluate outcomes of myeloablative non-TBI conditioning regimens for allogeneic HCT in B-ALLpatients at lower risk for relapse defined by absence of NGS-MRD (Clonoseq) of B-cell receptorrearrangements (BCR) just prior to HCT...Mismatched related/haploidentical grafts received post-transplant cyclophosphamide orTCRαβ/CD19 depletion according to institutional preference...Of patientsenrolled, 33% were White/Non-Hispanic, 37% Hispanic, 12% Black or African American, and..."
Biomarker • Clinical • IO biomarker • Minimal residual disease • Next-generation sequencing • Residual disease • Acute Graft versus Host Disease • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • CNS Disorders • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Transplantation
August 31, 2026
Thiotepa-busulfan-fludarabine conditioning in allogeneic hematopoietic stem cell transplantation for blast-phase myeloproliferative neoplasm
(PubMed, Rinsho Ketsueki)
- "This case suggests that a multidisciplinary treatment strategy incorporating TBF conditioning may improve relapse-free survival in patients with BP-MPN."
Journal • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Essential Thrombocythemia • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Leukemia • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Otorhinolaryngology • Thrombocytosis • Transplantation
August 05, 2026
Acalabrutinib in relapsed/refractory primary and secondary central nervous system lymphomas
(EANO 2026)
- "Pts received a median of 2.5 (1-3) prior treatments, including MTX (9), CD19 directed CAR T cell therapy (5), cytarabine (5), radiation (4), etoposide (3), rituximab (3), temozolomide (3), thiotepa-based autologous transplantation (2). Acalabrutinib at a higher dose level of 200 mg oral twice daily was well-tolerated in CNSL patients. The overall response rate was modest, likely reflecting the heavily pre-treated population, including 5/9 patients who had progressed after CAR T-cell therapy. Analysis of genomic features and CSF PKs may provide insight into efficacy."
Brain Cancer • Chronic Lymphocytic Leukemia • CNS Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Oncology • Primary Central Nervous System Lymphoma • Secondary Central Nervous System Lymphoma • Solid Tumor
November 04, 2025
Results of the primary end-point of the LOC-R01: A randomized phase II Study of lenalidomide and ibrutinib in association with rituximab-methotrexate procarbazine vincristin (R-MPV) as a targeted induction treatment for patients aged 18 to 65 with a newly diagnosed primary central nervous system lymphoma (PCNSL)
(ASH 2025)
- P1/2 | "Background : With standard high-dose methotrexate (HDMTX) and cytarabine (HDAraC)-basedinductions, half of the patients achieved complete response (CR)...Responders received two cycles of HDAraCfollowed by HD thiotepa-busulfan and ASCT... Both arms met the predetermined threshold of efficacy with 86% and 82% of CR/CRurates in the lenalidomide and ibrutinib arm, respectively. R-MPV plus a BTK-inhibitor or animmunomodulatory drug constitutes an interesting first-line induction for PCNSL patients up to 65 years.Correlation of patient, disease and lymphoid subpopulations characteristics with response in eachtreatment arm will be explored to guide the choice of the targeted therapy. Ancillary studies regardingthe prognostic impact of baseline and end of induction levels of cytokines in the CSF, ctDNA inplasma/CSF and radiomic features, as biomarkers of response, are planned."
Clinical • IO biomarker • P2 data • CNS Disorders • CNS Lymphoma • Hematological Malignancies • Hepatology • Lymphoma • Mental Retardation • Non-Hodgkin’s Lymphoma • Primary Central Nervous System Lymphoma • Steven-Johnson Syndrome • CD4 • ICOS • PD-1
September 05, 2026
Conditioning Intensity and Outcomes in Pediatric HLA-Identical Transplant for Sickle Cell Disease: Comparison of MAC, RIC, and NMA.
(PubMed, Transplant Cell Ther)
- P2 | "NMA conditioning with alemtuzumab and low-dose TBI substantially reduces toxicity compared with MAC and RIC. Although some patients require second HCT to improve donor engraftment, overall outcomes support this NMA approach as an option for children and adolescents with SCD."
Journal • Chronic Graft versus Host Disease • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Immunology • Pediatrics • Sickle Cell Disease • Transplantation
September 11, 2026
Characteristics and Outcomes of Central Nervous System Multiple Myeloma
(IMS 2026)
- "Pts received a median of 4 (R:1-16) doses of IT therapy, 4/5 received thiotepa (2 in combination with methotrexate & cytarabine, & mercaptopurine respectively), & 1 received methotrexate alone. Systemic treatment included combination of proteasome inhibitors, immunomodulatory drugs, anti-CD38 antibodies, selinexor, & other novel therapeutics (1 received ciltacabtagene autoleucel (ciltacel), & another received talquetamab followed by teclistamab)...Pts with CSF clearance (2/5) had received ciltacel & VD-PACE, while 2/5 with radiographic response had received ciltacel & daratumumab-pomalidomide... CNS myeloma pts had a dismal prognosis. All pts relapsed within 30m of ASCT, pointing to functionally high-risk disease. This highlights the need for increased awareness for early identification & CNS directed therapies."
CNS Disorders • Hematological Malignancies • Leukemia • Multiple Myeloma • Plasma Cell Leukemia
April 10, 2024
Myeloablative Vs. Non-Myeloablative Consolidation for Primary Central Nervous System Lymphoma: Results of Alliance 51101.
(PubMed, Blood Adv)
- P2 | "Patients, age 18-75 years, were randomly assigned in a 1:1 manner to induction therapy (methotrexate, temozolomide, rituximab and cytarabine) followed by consolidation with either thiotepa plus carmustine and autologous stem cell rescue versus induction followed by non-myeloablative, infusional etoposide plus cytarabine (EA) The primary endpoint was progression-free survival (PFS). Both consolidative strategies yielded encouraging efficacy and similar toxicity profiles. Clinicaltrials.gov (NCT01511562)."
Journal • Bone Marrow Transplantation • CNS Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Transplantation
September 01, 2026
Successful Autologous Hematopoietic Stem Cell Transplantation in HIV-Associated Primary CNS Lymphoma
(SOHO 2026)
- "He underwent thiotepa/carmustine-conditioned auto-HSCT with neutrophil engraftment at day +18 and platelet recovery at day +19. This case demonstrates that auto-HSCT is feasible and effective in HIV-associated PCNSL despite profound baseline immunosuppression. It challenges historical contraindications to transplantation in PLWH with CNS disease and supports reconsideration of eligibility criteria for curative-intent therapies and clinical trials. ART: antiretroviral therapy; auto-HSCT: autologous hematopoietic stem cell transplantation; CD: cluster of differentiation; CNS: central nervous system; DLBCL: diffuse large B-cell lymphoma; HIV: human immunodeficiency virus; MATRix: methotrexate, cytarabine, thiotepa, and rituximab; PCNSL: primary CNS lymphoma; PLWH: people living with HIV; R-MPV: rituximab plus high-dose methotrexate, procarbazine, and vincristine."
B Cell Lymphoma • CNS Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Primary Central Nervous System Lymphoma • CD34
September 12, 2026
Efficacy and Safety of Intraventricular Thiotepa in Combination With Methotrexate for Leptomeningeal Metastasis From Breast Cancer (NJMU-BC04): A Single-Arm Phase II Study.
(PubMed, MedComm (2020))
- "The exploratory study indicated that two proteins in CSF and four plasma proteins were significantly associated with iORR. Overall, the regimen was feasible and demonstrated a manageable safety profile, with a preliminary signal of clinical benefit warranting further investigation."
Journal • P2 data • Breast Cancer • Hematological Disorders • Leukopenia • Oncology • Solid Tumor • Thrombocytopenia
November 03, 2023
Moderate Incidence but Striking Correlation with TBI of Secondary Malignancies after HSCT in Children with ALL: Long-Term Follow-up from the Prospective International BFM- and Forum-Trials
(ASH 2023)
- "Only pts with contraindications for TBI received chemo-conditioning, which consisted of i.v. busulfan, cyclophosphamide and etoposide. In the FORUM trial pts >4y of age were randomized to receive either TBI- or chemo-based conditioning regimens; pts 2 y of age at HSCT (94%, n=2024). TBI-based conditioning was applied in 64% (n=1429), while 34% (n=722) of pts received chemo-conditioning."
Clinical • Acute Graft versus Host Disease • Acute Lymphocytic Leukemia • Basal Cell Carcinoma • Bone Marrow Transplantation • Brain Cancer • Breast Cancer • Chronic Graft versus Host Disease • CNS Tumor • Endocrine Cancer • Glioblastoma • Graft versus Host Disease • Hematological Malignancies • Leukemia • Non-melanoma Skin Cancer • Oncology • Pediatrics • Solid Tumor • Squamous Cell Carcinoma • Thyroid Gland Carcinoma • Transplantation • TP53
September 08, 2026
Comparison of Thiotepa, Busulfan, and Cyclophosphamide Versus Busulfan and Thiotepa Conditioning Regimens in Autologous Stem Cell Transplantation for Primary CNS Lymphoma: A Multicenter Retrospective Analysis.
(PubMed, Hematol Oncol)
- "We retrospectively analyzed patients aged ≤ 60 years who underwent ASCT with thiotepa-busulfan-cyclophosphamide (TBC) or busulfan-thiotepa (BuTT) conditioning after high-dose methotrexate-based induction at two Korean institutions between 2015 and 2023...In younger patients with PCNSL, TBC demonstrated a trend toward more durable disease control than BuTT, with manageable toxicity. As the thiotepa dose represents the major distinction between regimens, dose-intensified thiotepa strategies with adjusted partner agents merit further prospective investigation."
Clinical • Journal • Retrospective data • CNS Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Primary Central Nervous System Lymphoma • Transplantation
September 02, 2026
SCDHCT: Reduced Intensity Transplantation for Severe Sickle Cell Disease
(clinicaltrials.gov)
- P2 | N=46 | Active, not recruiting | Sponsor: St. Jude Children's Research Hospital | Suspended ➔ Active, not recruiting
Enrollment closed • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • Transplantation
May 04, 2023
CONSOLIDATIVE HCT-ASCT IS SUPERIOR TO NON-MYELOABLATIVE CHEMO-IMMUNOTHERAPY IN NEWLY-DIAGNOSED PCNSL - UPDATED RESULTS OF THE RANDOMIZED PHASE III MATRIX/IELSG43 TRIAL
(ICML 2023)
- P3 | "Induction comprised four cycles MATRix (rituximab 375 mg/m2/d days(d) 0,5; methotrexate 3.5 g/m2 d1; cytarabine 2 × 2 g/m2/d d2,3; thiotepa 30 mg/m2 d4, every three weeks...Arm A consisted of two cycles R-DeVIC (375 mg/m2 d0; dexamethasone 40 mg/d d1–3; etoposide 100 mg/m2/d d1–3; ifosfamide 1500 mg/m2/d d1–3; carboplatin 300 mg/m2 d1); Arm B, consisted of HDC-ASCT (BCNU 400 mg/m2 (d-6) and thiotepa 2 × 5 mg/kg/d d-5,-4))... 368 pts were registered between July 2014 and August 2019, 230/346 pts (67%) were randomly assigned to arm A and arm B, respectively. 116 patients discontinued induction treatment, mainly due to toxicity (15%) or progressive disease (12%). Median age of the randomized pts was 59 years (range 21–70) with 22% being ≥65 years."
Clinical • P3 data • CNS Lymphoma
July 15, 2022
Radiotherapy or Autologous Stem-Cell Transplantation for Primary CNS Lymphoma in Patients Age 60 Years and Younger: Long-Term Results of the Randomized Phase II PRECIS Study.
(PubMed, J Clin Oncol)
- "Patients were treated with high-dose methotrexate-based induction chemotherapy followed by whole-brain radiotherapy (WBRT) or high-dose chemotherapy (thiotepa-busulfan-cyclophosphamide) with autologous stem-cell transplantation (ASCT)...Balance (52% v 10%, P ≤ 0.001) and neurocognition (64% v 13%, P < .001) significantly deteriorated after WBRT compared with ASCT during the follow-up. This study shows that 40 Gy WBRT should be avoided in first-line treatment because of its neurotoxicity and suboptimal efficacy in reducing relapses while ASCT appears to be highly efficient in preventing relapses."
Journal • P2 data • Bone Marrow Transplantation • CNS Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Transplantation
September 11, 2026
Clinical Outcomes and Predictors of Survival in Central Nervous System (CNS) Multiple Myeloma: A Retrospective Cohort Study
(IMS 2026)
- "Sixteen patients received combination IT therapy (methotrexate, cytarabine and hydrocortisone) and 3 received IT thiotepa. CNS-MM remains an aggressive manifestation of MM with poor survival outcomes. Although patients receiving CAR T-cell therapy and/or BsAbs in our cohort demonstrated numerically longer overall survival, these findings did not reach statistical significance, likely due to the small sample size. Effect of novel immunotherapies in patients with CNS MM may require further investigation in larger cohort studies."
Clinical data • Retrospective data • Hematological Malignancies • Multiple Myeloma
November 04, 2025
Allogeneic HSC and regulatory T cell (Orca-T) engineered cell therapy following reduced intensity conditioning: Results of a single center Phase 1 study
(ASH 2025)
- P3 | "Fourteen subsequent patients received 10mg/kg of thiotepa instead of melphalan,with the same dosing of fludarabine and 4 Gy TBI. These early results demonstrate that Orca-T is a safe and feasible transplant strategy forRIC conditioning. The trial achieved the goal of identifying a lympho-depletive RIC conditioning regimentolerated in the outpatient setting. Patients showed robust engraftment and donor T cell chimerism."
Clinical • P1 data • Acute Graft versus Host Disease • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Hepatology • Immunology • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm
September 04, 2026
NCI-2018-01752: Naive T Cell Depletion for Preventing Chronic Graft-versus-Host Disease in Children and Young Adults With Blood Cancers Undergoing Donor Stem Cell Transplant
(clinicaltrials.gov)
- P2 | N=45 | Active, not recruiting | Sponsor: Fred Hutchinson Cancer Center | Recruiting ➔ Active, not recruiting | N=68 ➔ 45
Enrollment change • Enrollment closed • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Burkitt Lymphoma • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Lymphoma • Mast Cell Leukemia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Non-Hodgkin’s Lymphoma • Oncology • Transplantation • HLA-B • HLA-C • HLA-DRB1
November 03, 2023
Genetic Engineering of Hematopoietic Progenitor Stem Cells for Targeted IFN-α Immunotherapy Reprogramming the Solid Tumor Microenvironment: A First-in-Man Study in Glioblastoma Multiforme (NCT03866109)
(ASH 2023)
- P1/2 | "Autologous CD34+ HSPC are mobilized with lenograstim and plerixafor, collected by apheresis, purified and ex vivo modified with a lentiviral vector. So far, up to 3 million Temferon cells/kg have been co-administered with a fixed dose of non-manipulated CD34+ supporter cells following a sub-myeloablative conditioning regimen (Thiotepa + BCNU or Busulfan or Busulfan alone)... These data show that Temferon is safe and biologically active at the tumor site and favors anti-tumor immunity. The results provide initial evidence of Temferon's potential to modulate the TME of GBM patients and to counteract disease progression and improve the survival of uMGMT GBM patients."
Biomarker • IO biomarker • Tumor microenvironment • Brain Cancer • CNS Tumor • Gene Therapies • Glioblastoma • Oncology • Solid Tumor • CD34 • CD8 • IFNA1 • MGMT • PTPRC
November 23, 2022
Effects on Survival of Non-Myeloablative Chemoimmunotherapy Compared to High-Dose Chemotherapy Followed By Autologous Stem Cell Transplantation (HDC-ASCT) As Consolidation Therapy in Patients with Primary CNS Lymphoma - Results of an International Randomized Phase III Trial (MATRix/IELSG43)
(ASH 2022)
- P3 | "Induction consisted of 4 cycles of MATRix regimen (rituximab 375 mg/m2/d days 0 & 5; methotrexate 3.5 g/m2 day 1; cytarabine 2 × 2 g/m2/d days 2 & 3; thiotepa 30 mg/m2 day 4, every 21 days)...Pts achieving at least partial response (PR) after completion of induction were randomly allocated to either arm A with two courses of R-DeVIC regimen (375 mg/m2 day 0; dexamethasone 40 mg/d days 1 to 3; etoposide 100 mg/m2/d days 1 to 3; ifosfamide 1500 mg/m2/d days 1 to 3; carboplatin 300 mg/m2 day 1); or arm B, consisting of HDC with BCNU 400 mg/m2 (day -6) and thiotepa 2 x 5 mg/kg/d days -5 & -4) followed by ASCT... This international randomized phase III trial demonstrates that consolidation with HDC-ASCT results in significantly better outcome than non-myeloablative chemoimmunotherapy. This comes along without any measurable negative effect on neurocognitive functions and with an excellent risk-to-benefit ratio. HDC-ASCT is the standard consolidation..."
Clinical • Late-breaking abstract • P3 data • Bone Marrow Transplantation • CNS Lymphoma • Hematological Malignancies • Human Immunodeficiency Virus • Infectious Disease • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Transplantation
November 04, 2025
Addition of thiotepa to a busulphan based conditioning regimen does not improve survival in patients allografted for acute myeloid leukaemia and myelodysplasia: Results of the UK impact cosi trial
(ASH 2025)
- "Thiotepa (Thio)is an alkylating agent whose addition to a busulphan (Bu)/fludarabine (Flu) conditioning regimen hasbeen shown in retrospective studies to improve survival in patients transplanted for AML using bothmatched unrelated and haploidentical donors, consequent upon a reduction in post-transplant relapse.As a result, Flu/Bu/Thio conditioning regimens are increasingly used in patients allografted for high riskAML despite the absence of prospective randomised trials supporting this practice...All patients received ciclosporin/ATG-based GVHD prophylaxis... This prospective randomised trial demonstrates that the addition of Thio to either a Flu/Bubased MAC or RIC regimen does not improve survival in patients allografted for AML or MDS and wasassociated with an increased TRM in both settings. Further prospective studies examining the ability ofThio to reduce the risk of disease relapse in high risk patients whilst at the same time limiting transplanttoxicity are merited."
Clinical • Acute Myelogenous Leukemia • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome
May 15, 2022
Long-term efficacy, safety and neurotolerability of MATRix regimen followed by autologous transplant in primary CNS lymphoma: 7-year results of the IELSG32 randomized trial.
(PubMed, Leukemia)
- "Enrolled patients were randomly assigned to receive methotrexate-cytarabine (arm A), or methotrexate-cytarabine-rituximab (B), or methotrexate-cytarabine-thiotepa-rituximab (MATRix; arm C). A second randomization allocated patients with responsive/stable disease to whole-brain irradiation (WBRT) or carmustine-thiotepa-conditioned autologous transplantation (ASCT)...Importantly, MATRix and ASCT did not result in higher non-relapse mortality or second tumors incidence. Patients who received WBRT experienced impairment in attentiveness and executive functions, whereas patients undergoing ASCT experienced improvement in these functions as well as in memory and quality of life."
Journal • Alzheimer's Disease • CNS Disorders • CNS Lymphoma • Cognitive Disorders • Hematological Malignancies • Human Immunodeficiency Virus • Infectious Disease • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Transplantation
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