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DRUG:

thiotepa

i
Other names: DSP-1958
Company:
Generic mfg.
Drug class:
DNA synthesis inhibitor, Alkylating agent
Related drugs:
1d
Itacitinib for the Prevention of Graft Versus Host Disease (clinicaltrials.gov)
P1, N=30, Active, not recruiting, M.D. Anderson Cancer Center | Trial completion date: Feb 2027 --> Apr 2025
Trial completion date
|
cyclophosphamide • fludarabine IV • thiotepa • busulfan • itacitinib (INCB039110)
4d
US Study of ECT-001-CB in Pediatric and Young Adult Patients With High-Risk Myeloid Malignancies (clinicaltrials.gov)
P1/2, N=12, Recruiting, ExCellThera inc. | Trial completion date: Dec 2025 --> Jun 2026 | Trial primary completion date: Dec 2023 --> Jun 2026
Trial completion date • Trial primary completion date
|
HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • CD34 (CD34 molecule)
|
cyclophosphamide • clofarabine • fludarabine IV • thiotepa • busulfan • ECT-001-CB
4d
Reduced Intensity Allogeneic HCT in Advanced Hematologic Malignancies w/T-Cell Depleted Graft (clinicaltrials.gov)
P1, N=40, Recruiting, Stanford University | Trial completion date: Jun 2024 --> Dec 2025 | Trial primary completion date: Jun 2024 --> Dec 2025
Trial completion date • Trial primary completion date • Metastases
|
HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • HLA-B (Major Histocompatibility Complex, Class I, B) • HLA-C (Major Histocompatibility Complex, Class I, C)
|
Jakafi (ruxolitinib) • cyclophosphamide • melphalan • fludarabine IV • thiotepa • Neupogen (filgrastim) • plerixafor
7d
HeadStart4: Newly Diagnosed Children (<10 y/o) With Medulloblastoma and Other CNS Embryonal Tumors (clinicaltrials.gov)
P4, N=250, Active, not recruiting, Nationwide Children's Hospital | Recruiting --> Active, not recruiting
Enrollment closed
|
cisplatin • carboplatin • cyclophosphamide • etoposide IV • vincristine • thiotepa • methotrexate IV
7d
New P2 trial • Post-transplantation
|
cyclophosphamide • clofarabine • fludarabine IV • thiotepa • busulfan
9d
Itacitinib for the Prevention of Graft Versus Host Disease (clinicaltrials.gov)
P1, N=30, Active, not recruiting, M.D. Anderson Cancer Center | Recruiting --> Active, not recruiting
Enrollment closed
|
cyclophosphamide • fludarabine IV • thiotepa • busulfan • itacitinib (INCB039110)
14d
Venetoclax to Improve Outcomes of Fractionated Busulfan Regimen in Patients With High-Risk AML and MDS (clinicaltrials.gov)
P2, N=103, Active, not recruiting, M.D. Anderson Cancer Center | Recruiting --> Active, not recruiting
Enrollment closed
|
KRAS (KRAS proto-oncogene GTPase) • NRAS (Neuroblastoma RAS viral oncogene homolog) • DNMT3A (DNA methyltransferase 1) • NF1 (Neurofibromin 1) • RUNX1 (RUNX Family Transcription Factor 1) • ASXL1 (ASXL Transcriptional Regulator 1) • PTPN1 (Protein Tyrosine Phosphatase Non-Receptor Type 1)
|
TP53 mutation • KRAS mutation • NRAS mutation • RAS mutation
|
Venclexta (venetoclax) • cladribine • fludarabine IV • thiotepa • busulfan
28d
Enrollment open • Enrollment change
|
HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • CD34 (CD34 molecule) • HLA-B (Major Histocompatibility Complex, Class I, B)
|
cyclophosphamide • fludarabine IV • thiotepa • dilanubicel (NLA101)
1m
Enrollment closed
|
FLT3 (Fms-related tyrosine kinase 3) • RUNX1 (RUNX Family Transcription Factor 1) • KMT2A (Lysine Methyltransferase 2A) • ETV6 (ETS Variant Transcription Factor 6) • WT1 (WT1 Transcription Factor) • CREBBP (CREB binding protein) • NUP98 (Nucleoporin 98 And 96 Precursor 2) • NSD1 (Nuclear Receptor Binding SET Domain Protein 1) • HOXA9 (Homeobox A9) • NUP214 (Nucleoporin 214) • CBFA2T3 (CBFA2/RUNX1 Partner Transcriptional Co-Repressor 3) • GLIS2 (GLIS Family Zinc Finger 2) • KAT6A (Lysine Acetyltransferase 6A) • KDM5A (Lysine Demethylase 5A) • DEK (DEK Proto-Oncogene) • AFDN (Afadin, Adherens Junction Formation Factor) • MLLT10 (MLLT10 Histone Lysine Methyltransferase DOT1L Cofactor)
|
FLT3-ITD mutation • WT1 mutation • MLL fusion • NUP98-NSD1 fusion
|
cyclophosphamide • Blincyto (blinatumomab) • melphalan • fludarabine IV • thiotepa • Neupogen (filgrastim)
1m
A Modified Dose of Rabbit Anti-thymocyte Globulin (rATG) in Children and Adults Receiving Treatment to Help Prepare Their Bodies for a Bone Marrow Transplant (clinicaltrials.gov)
P2, N=59, Recruiting, Memorial Sloan Kettering Cancer Center | Trial completion date: Apr 2024 --> Apr 2025 | Trial primary completion date: Apr 2024 --> Apr 2025
Trial completion date • Trial primary completion date
|
TP53 (Tumor protein P53) • HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • CD34 (CD34 molecule) • CD4 (CD4 Molecule)
|
TP53 mutation
|
cyclophosphamide • melphalan • fludarabine IV • thiotepa • busulfan
2ms
New P1 trial
|
melphalan • fludarabine IV • thiotepa • Orca-T
2ms
Umbilical Cord Blood Transplant, Cyclophosphamide, Fludarabine, and Total-Body Irradiation in Treating Patients With Hematologic Disease (clinicaltrials.gov)
P2, N=135, Active, not recruiting, Fred Hutchinson Cancer Center | Trial completion date: Dec 2025 --> Dec 2024
Trial completion date
|
HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • HLA-B (Major Histocompatibility Complex, Class I, B)
|
cyclophosphamide • fludarabine IV • thiotepa • cyclosporin A microemulsion
2ms
Palifermin With Leuprolide Acetate for the Promotion of Immune Recovery Following Total Body Irradiation Based T-Cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation (clinicaltrials.gov)
P2, N=82, Active, not recruiting, Memorial Sloan Kettering Cancer Center | Trial completion date: Dec 2023 --> Dec 2024 | Trial primary completion date: Dec 2023 --> Dec 2024
Trial completion date • Trial primary completion date
|
CD34 (CD34 molecule) • CD4 (CD4 Molecule)
|
Chr t(15;17)
|
cyclophosphamide • Firmagon (degarelix) • thiotepa • leuprolide acetate for depot suspension • Kepivance (palifermin)
2ms
Trial completion date • Trial primary completion date
|
Jakafi (ruxolitinib) • cyclophosphamide • fludarabine IV • mesna • thiotepa • busulfan • cyclosporine
2ms
Evidence-based management of primary and secondary CNS lymphoma. (PubMed, Semin Hematol)
Intensive high-dose methotrexate-containing induction followed by consolidation with autologous stem cell transplantation with thiotepa-based conditioning is recommended for patients who are fit...Data regarding the efficacy of chimeric antigen receptor T-cells therapy is emerging, and concerns regarding greater toxicity have not been realized. Future areas of prospective studies include the identification of those at high risk of developing CNS lymphoma, management in elderly or frail patients as well as incorporating novel agents into regimens, particularly for those with chemoresistant disease.
Journal
|
CRBN (Cereblon)
|
methotrexate • thiotepa
2ms
Reduced-Intensity Conditioning (RIC) and Myeloablative Conditioning (MAC) for HSCT in AML/MDS (clinicaltrials.gov)
P2, N=22, Completed, Randy Windreich | Active, not recruiting --> Completed | Trial completion date: Nov 2023 --> Apr 2023 | Trial primary completion date: Nov 2023 --> Apr 2023
Trial completion • Trial completion date • Trial primary completion date
|
FLT3 (Fms-related tyrosine kinase 3) • CD34 (CD34 molecule)
|
FLT3 positive
|
Campath (alemtuzumab) • melphalan • fludarabine IV • hydroxyurea • thiotepa • busulfan
2ms
New P3 trial
|
ALK (Anaplastic lymphoma kinase) • TERT (Telomerase Reverse Transcriptase) • MYCN (MYCN Proto-Oncogene BHLH Transcription Factor)
|
cisplatin • carboplatin • temozolomide • doxorubicin hydrochloride • cyclophosphamide • etoposide IV • irinotecan • vincristine • daunorubicin • topotecan • melphalan • thiotepa • Qarziba (dinutuximab beta) • Unituxin (dinutuximab) • dexrazoxane
2ms
Thiotepa Plus Fludarabine+ Melphalan as the Preparative Regime for Alternative Donor Transplantation (clinicaltrials.gov)
P2, N=40, Active, not recruiting, Case Comprehensive Cancer Center | Trial completion date: Dec 2025 --> Jun 2025 | Trial primary completion date: Dec 2023 --> Jun 2024
Trial completion date • Trial primary completion date
|
FLT3 (Fms-related tyrosine kinase 3)
|
FLT3 mutation
|
fludarabine IV • thiotepa • captisol-enabled melphalan
3ms
CD34+ Transplants for Leukemia and Lymphoma (clinicaltrials.gov)
P2, N=100, Not yet recruiting, Baptist Health South Florida | Trial completion date: Dec 2030 --> Mar 2031
Trial completion date
|
CD34 (CD34 molecule)
|
cyclophosphamide • melphalan • fludarabine IV • thiotepa • busulfan
3ms
New P1/2 trial
|
melphalan • fludarabine IV • thiotepa • busulfan • plerixafor
3ms
Unrelated and Haploidentical Peripheral Stem Cell Transplant with Tcrαβ/CD19 Depletion for the Treatment of Pediatric Patients with Inborn Errors of Immunity (TCT-ASTCT-CIBMTR 2024)
Conditioning was myeloablative busulfan with pharmacokinetic monitoring and fludarabine +/- cyclophosphamide/thiotepa or a reduced intensity melphalan-based regimen with fludarabine. All patients received serotherapy with ATG or alemtuzumab. Post-PSCT immune suppression was none or mycophenolate mofetil (MMF) and/or a calcineurin inhibitor (CNI)... 41 patients with a variety of IEIs were included (Figure 1). At a median follow-up of 2.5 years, OS was 85% (95% CI 75-97%) and EFS 80% (95% CI 69-94%), but was variable by underlying disease (Figure 2). Three patients experienced graft failure (7.3%), two requiring a second transplant and one receiving donor lymphocyte infusion only.
Clinical
|
CD4 (CD4 Molecule)
|
cyclophosphamide • Campath (alemtuzumab) • melphalan • fludarabine IV • thiotepa • busulfan
3ms
Excellent Outcomes Using a Non-Myeloablative Conditioning with Thiotepa and Post -Transplant Cyclophosphamide in Adults Undergoing a Matched Related Donor Bone Marrow Transplant for Severe Sickle Cell Disease (TCT-ASTCT-CIBMTR 2024)
The conditioning regimen included: rabbit anti-thyroglobulin (ATG), Thiotepa, Fludarabine, Cyclophosphamide, and total body irradiation (TBI). GVHD prophylaxis included PTCy, mycophenolate mofetil (MMF), and Sirolimus, Figure 3...The filgrastim mobilized stem cell source was PB in 54.5% and BM in 45.5%... This result shows excellent outcomes using Thiotepa based conditioning with PTCy for MSD or MMRD for adults with SCD. The toxicity was low, with no mortality or chronic GVHD, and well tolerated with a second transplant. The optimal donor engraftment (>90% donor chimerism) will help mitigate against the risk of secondary myeloid neoplasm.
Clinical • Post-transplantation
|
CD34 (CD34 molecule)
|
cyclophosphamide • sirolimus • fludarabine IV • thiotepa • Neupogen (filgrastim)
3ms
Car-T Pre-HSCT and Prophylactic Haplo-DLI Post-HSCT and Tcrαβ-Depleted Haploidentical Stem Cell Transplantation: Decreased the Relapse Rate and Improve the Transplant Outcome in Pediatric R/R-ALL (TCT-ASTCT-CIBMTR 2024)
The conditioning consisted of Cyclophosphamide (1 × 50 mg/kg, days −10, -3 to -2), Fudarabine (1 × 40 mg/m2, days -7 to -4), Thiotepa (2 × 5 mg/kg, day -4), Bu ( total 90 mg/m2, days -7 to -5) , Anti-thymocyte globulin (ATG-F) (1 × 15 mg/kg,days -9 to -8 ). With a median follow-up of 431 (range, 16-1062) days for surviving patients. The median time to neutrophil and platelet engraftment was 17 (range, 10–22) and 9 (range, 6–12) days, respectively. Three children experienced primary graft failure.
Clinical
|
CD22 (CD22 Molecule) • CD7 (CD7 Molecule)
|
cyclophosphamide • thiotepa
3ms
Clinical Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation Using Busulfan/Cyclophosphamide and Melphalan or Thiotepa As Conditioning Regimen in 37 Patients with Acute Megakaryoblastic Leukemia (ASH 2023)
The conditioning regimens administered were modified Bu/Cy (fludarabine 30 mg/m 2/d×5d; cytarabine 2g/m 2/d×5d; Bu 3...8g/m 2/d×2d; etoposide 100mg/m 2/d×2d; antithymocyte globulin 1...Cyclosporine A (CSA)+ mycophenolate mofetil (MMF)+ short methotrexate (sMTX) were used as graft-versus-host disease (GVHD) prophylaxis...Therefore, it is strongly recommended that allo-HSCT is performed in CR status. However, to corroborate these findings, it is essential to conduct long-term, large-scale clinical research across multiple centers.
Clinical • Clinical data
|
NRAS (Neuroblastoma RAS viral oncogene homolog) • JAK2 (Janus kinase 2) • ASXL1 (ASXL Transcriptional Regulator 1) • TET2 (Tet Methylcytosine Dioxygenase 2) • PTPN11 (Protein Tyrosine Phosphatase Non-Receptor Type 11) • WT1 (WT1 Transcription Factor) • NUP98 (Nucleoporin 98 And 96 Precursor 2) • CD34 (CD34 molecule) • CBFA2T3 (CBFA2/RUNX1 Partner Transcriptional Co-Repressor 3) • GATA1 (GATA Binding Protein 1) • GLIS2 (GLIS Family Zinc Finger 2) • KDM5A (Lysine Demethylase 5A)
|
WT1 mutation
|
cytarabine • cyclophosphamide • etoposide IV • methotrexate • melphalan • fludarabine IV • thiotepa • busulfan
3ms
Phase II, Single-Center, Prospective Study of Intrathecal Thiotepa for the Prophylaxis of Secondary Central Nervous System Involvement in Highly Aggressive B-Cell Lymphoma (ASH 2023)
Evidence supporting the use of intravenous methotrexate (MTX) or arabinoside (AraC) in high-risk patients is growing, however, the use of other agents remains to be defined...All patients are need to receive IT thiotepa 10 mg and dexamethasone 5 mg on day 1 of each cycle of chemotherapy for at least 4 cycles...6%) patients received rituximab, cyclophosphamide, doxorubicin, vincristine, prednisone (RCHOP), 5 (18...1%) patients received BTK inhibitor, lenalidomide with rituximab (ZR2) followed by RCHOP, and 4 (14...ConclusionProphylactic intrathecal thiotepa is an effective and side-effects manageable measure for preventing secondary central nervous system involvement in high-risk DLBCL. Longer follow-up and larger-scale prospective trial is needed to testify this measure.
Clinical • P2 data • IO biomarker
|
TP53 (Tumor protein P53) • MYC (V-myc avian myelocytomatosis viral oncogene homolog) • BCL2 (B-cell CLL/lymphoma 2) • BCL6 (B-cell CLL/lymphoma 6)
|
TP53 deletion
|
Rituxan (rituximab) • lenalidomide • doxorubicin hydrochloride • cyclophosphamide • vincristine • thiotepa
3ms
Methotrexate in Combination with Orelabrutinib, Thiotepa, and Rituximab (R-MTO) Followed By Autologous Stem-Cell Transplant for Primary Central Nervous System Lymphoma Treatment (ASH 2023)
The HCT regimen involved busulfan 3. 2 mg/kg on days -5, -4, thiotepa 250 mg/m 2 on days -7 and -6, cytarabine 2g/m 2 q12h on days -3 and -2, followed by reinfusion of stem cells on day 0... The R-MTO induction treatment has demonstrated notable efficacy in achieving higher response rates among patients with PCNSL, and the associated adverse events are manageable.
Combination therapy
|
TP53 (Tumor protein P53)
|
TP53 mutation
|
Rituxan (rituximab) • cytarabine • methotrexate • Yinuokai (orelabrutinib) • thiotepa • busulfan
3ms
Cord Blood Transplant With Dilanubicel for the Treatment of HIV Positive Hematologic Cancers (clinicaltrials.gov)
P2, N=10, Recruiting, Fred Hutchinson Cancer Center | Trial primary completion date: Nov 2022 --> Dec 2024
Trial primary completion date
|
MLL rearrangement • MLL rearrangement
|
cyclophosphamide • fludarabine IV • thiotepa • dilanubicel (NLA101)
3ms
TEAM (Thiotepa, Etoposide, Cytosar, Melphalan ) for AutoSCT in Lymphoma (clinicaltrials.gov)
P2, N=50, Active, not recruiting, Sheba Medical Center | Recruiting --> Active, not recruiting | Trial completion date: Dec 2022 --> Apr 2024 | Trial primary completion date: Jun 2022 --> Apr 2024
Enrollment closed • Trial completion date • Trial primary completion date
|
CD34 (CD34 molecule)
|
cytarabine • etoposide IV • melphalan • thiotepa
3ms
Intensive Conditioning With Thiotepa Combined With Bu/Flu/Ara-C in Allo-HSCT for Myeloid Malignancies With Extramedullary Involvement (clinicaltrials.gov)
P=N/A, N=50, Not yet recruiting, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine | Trial completion date: Nov 2027 --> Jan 2027
Trial completion date
|
HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • HLA-B (Major Histocompatibility Complex, Class I, B) • HLA-C (Major Histocompatibility Complex, Class I, C)
|
cytarabine • fludarabine IV • thiotepa • busulfan
3ms
Haploidentical (Half-matched) Related Donor Stem Cell Transplantation Using Killer Immunoglobulin-like Receptors in Addition to Normal Selection Factors to Determine the Best Donor (clinicaltrials.gov)
P=N/A, N=44, Completed, Memorial Sloan Kettering Cancer Center | Active, not recruiting --> Completed | Trial completion date: Aug 2024 --> Nov 2023 | Trial primary completion date: Aug 2024 --> Nov 2023
Trial completion • Trial completion date • Trial primary completion date
|
TP53 (Tumor protein P53) • FLT3 (Fms-related tyrosine kinase 3) • ABL1 (ABL proto-oncogene 1) • BCR (BCR Activator Of RhoGEF And GTPase) • RUNX1 (RUNX Family Transcription Factor 1) • ASXL1 (ASXL Transcriptional Regulator 1) • CRLF2 (Cytokine Receptor Like Factor 2) • IKZF1 (IKAROS Family Zinc Finger 1)
|
TP53 mutation • FLT3 mutation • RUNX1 mutation • ASXL1 mutation • IKZF1 mutation • MLL mutation
|
cyclophosphamide • melphalan • fludarabine IV • mesna • thiotepa • Neupogen (filgrastim)
3ms
NCI-2019-03188: Graft Versus Host Disease-Reduction Strategies for Donor Blood Stem Cell Transplant Patients With Acute Leukemia or Myelodysplastic Syndrome (MDS) (clinicaltrials.gov)
P2, N=120, Recruiting, Fred Hutchinson Cancer Center | Trial completion date: Aug 2026 --> Dec 2028 | Trial primary completion date: Aug 2025 --> Dec 2026
Trial completion date • Trial primary completion date • Post-transplantation
|
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)
|
cyclophosphamide • sirolimus • fludarabine IV • thiotepa • busulfan
3ms
HAPLOIDENTICAL ALLOGENIC TRANSPLANTATION IN HL R/R: EXPIRIENCE OF ONE SINGLE CENTER WITH T CELL DEPLETED TRANSPLANT (SIE 2023)
Conditioning consisted of ATG 1,5 mg/kg from day -13 to day -10, Treosulfan 12gr/sqm from -9 to –7, Fludarabine 30 mg/sqm from -6 to -2 and Thiotepa 5 mg/Kg on days -5 and -4. CMV IgG positive patients received prophylaxis with Ganciclovir during conditioning. All patients received Ambisome for fungal prophylaxis...Conclusions. The infusion of abT/CD19-depleted graft is a sure and effective option for heavely pretreated patients affected by HL R/R resulting into rapid donor hematopoietic engraftment and early expansion of donor-derived γδT lymphocytes, without life-threatening infectious complications and very low incidence of GVHD.
Clinical
|
CD34 (CD34 molecule)
|
fludarabine IV • thiotepa • Ovastat (treosulfan)
4ms
NCI-2017-02205: Infusion of Expanded Cord Blood Cells in Addition to Single Cord Blood Transplant in Treating Patients With Acute Leukemia, Chronic Myeloid Leukemia, or Myelodysplastic Syndromes (clinicaltrials.gov)
P2, N=15, Recruiting, Fred Hutchinson Cancer Center | Trial completion date: Oct 2025 --> Jun 2026 | Trial primary completion date: Oct 2023 --> Oct 2024
Trial completion date • Trial primary completion date
|
HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • CD34 (CD34 molecule) • HLA-B (Major Histocompatibility Complex, Class I, B)
|
cyclophosphamide • fludarabine IV • thiotepa • dilanubicel (NLA101)
4ms
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)
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.
IO biomarker
|
MGMT (6-O-methylguanine-DNA methyltransferase) • CD8 (cluster of differentiation 8) • CD34 (CD34 molecule) • PTPRC (Protein Tyrosine Phosphatase Receptor Type C) • IFNA1 (Interferon Alpha 1)
|
thiotepa • busulfan • Granocyte (lenograstim) • Temferon (Autologous CD34+ enriched HSPCs expressing interferon-alpha 2) • plerixafor
4ms
Excellent Outcomes of HLA Matched Related Donor Transplant for Adults with Severe Sickle Cell Disease Using a Non-Myeloablative Conditioning with Thiotepa and Post-Transplant Cyclophosphamide: Multi-Center International Experience (ASH 2023)
The conditioning regimen included (Figure3): rabbit anti-thymoglobulin (ATG) 4.5 mg/kg, thiotepa 10 mg/kg, fludarabine 150 mg/m2, cyclophosphamide 29 mg/kg and total body irradiation (TBI) 200 cGy. GVHD prophylaxis included PTCy 100 mg/kg, mycophenolate mofetil (MMF), and sirolimus. Stem cell source included bone marrow (BM) mobilized with filgrastim (5-10 μg/kg/d x 3-5 days) and peripheral blood (PB)... This result shows the superiority of Thiotepa based non-myeloablative conditioning and PTCy for MRD transplants in adults with severe SCD. It not only minimizes the toxicity and GVHD but provides optimal donor engraftment mitigating against risk of secondary myeloid neoplasms from mixed chimerism. This needs further validation in a larger cohort.
Clinical • Post-transplantation
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CD34 (CD34 molecule)
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cyclophosphamide • sirolimus • fludarabine IV • thiotepa • Neupogen (filgrastim)
4ms
[CANCELED] Outcome of Children with B-Cell Precursor Acute Lymphoblastic Leukemia (BCP-ALL) with Hypodiploidy or BCR-ABL1 Fusion Given Allogeneic Hematopoietic Stem Cell Transplantation (HSCT): Results from the Prospective Forum Study (ASH 2023)
Total body irradiation (TBI) + VP16 was administered to 42 and 50 pts in the HYPO and BCRABL groups, respectively, while 18 HYPO and 30 BCRABL pts received non-TBI conditioning (busulfan or treosulfan + fludarabine and thiotepa, as per study protocol)...Notably, 24 out of 143 pts (17%) with HYPO/BCRABL received immunotherapy before HSCT (blinatumomab, inotuzumab ozogamicin, and/or CAR-T cells)... Children receiving HSCT for treatment of BCP-ALL with either hypodiploidy or bcr-abl1 fusion showed outcomes comparable to BCP-ALL pts without these genetic lesions. CIR and EFS reached plateaus beyond 3 y post-HSCT, indicating that approximately two-thirds of these pts can be cured by HSCT with low rates of treatment-related mortality. Pts with BCRABL achieved favorable outcomes without the need for long-term or lifelong TKI therapy.
Clinical
|
ABL1 (ABL proto-oncogene 1) • BCR (BCR Activator Of RhoGEF And GTPase)
|
BCR-ABL1 fusion
|
Blincyto (blinatumomab) • Besponsa (inotuzumab ozogamicin) • fludarabine IV • thiotepa • busulfan • Ovastat (treosulfan)
4ms
Primary CNS Lymphoma in Latin America: A Grupo De Estudio Latinoamericano De Linfoproliferativos (GELL) Survey in 80 Cancer Centers in Latin America (ASH 2023)
Methotrexate (MTX) with Cytarabine was the preferred regimen for 36% of participants...The use of thiotepa-containing regimens has demonstrated improved survivals both as consolidation and in the R/R setting. Management of PCNSL is highly variable in LATAM. Access to targeted agents and novel therapies such as CAR-T cell therapy is limited. Moreover, most centers lack basic diagnostic approaches such as serum MTX level monitoring and ophthalmology evaluation.
PD(L)-1 Biomarker • IO biomarker
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PD-L1 (Programmed death ligand 1) • MYC (V-myc avian myelocytomatosis viral oncogene homolog) • BCL2 (B-cell CLL/lymphoma 2)
|
cytarabine • methotrexate • thiotepa
4ms
Budesonide Added to Post-Transplantation Cyclophosphamide, Tacrolimus and Mycophenolate Mofetil for Prevention of Graft-Versus-Host Disease (ASH 2023)
However, previous studies have not demonstrated their efficacy in preventing graft-versus-host disease (GVHD) when combined with the standard GVHD prophylaxis regimen consisting of calcineurin inhibitors and methotrexate...Thirty one pts received conditioning with fludarabine (160 mg/m2) + melphalan (100-140 mg/m2) + TBI (2-4Gy) or thiotepa 5 mg/kg...Among the pts who received treatment for aGVHD, the median cumulative systemic steroid exposure (in prednisone equivalents) was 1,318 mg (IQR 623-3,775) at D+100 and 1,325 mg (IQR 525-1,640) at D+180, with a median treatment duration of 40 days (IQR 25-110)... The addition of budesonide to PTCy-based GVHD prophylaxis regimen (PTCy-TMB) is feasible and appears to be effective in preventing severe GI aGVHD, without compromising transplant outcomes and immunologic reconstitution.
Post-transplantation
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CD8 (cluster of differentiation 8) • CD4 (CD4 Molecule) • NCAM1 (Neural cell adhesion molecule 1)
|
cyclophosphamide • methotrexate • melphalan • fludarabine IV • thiotepa
4ms
Optimizing Outcomes with Myeloablative Conditioning in Older Patients: Efficacy and Safety of Precision Engineered Orca-T in Patients > 55 Years Old with Hematologic Malignancies (ASH 2023)
Patients received a myeloablative regimen consisting of intravenous busulfan, fludarabine, and thiotepa (BFT) prior to Orca-T, followed by single-agent GvHD prophylaxis with tacrolimus, and had an 8/8 related or unrelated matched donor. Remarkably, non-relapse mortality was zero at 1 year with excellent overall survival , suggesting that this regimen could potentially improve outcomes for older patients. An ongoing Phase 3 trial evaluating Orca-T vs. standard of care is currently enrolling throughout the United States.
Clinical
|
HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1)
|
fludarabine IV • thiotepa • busulfan • Orca-T
4ms
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
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TP53 (Tumor protein P53)
|
TP53 mutation
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cyclophosphamide • etoposide IV • fludarabine IV • thiotepa • busulfan • Ovastat (treosulfan)
4ms
Trial completion date • Surgery
|
cisplatin • carboplatin • cyclophosphamide • etoposide IV • vincristine • leucovorin calcium • thiotepa • Neupogen (filgrastim)
4ms
Intensive Conditioning With Thiotepa Combined With Bu/Flu/Ara-C in Allo-HSCT for Myeloid Malignancies With Extramedullary Involvement (clinicaltrials.gov)
P=N/A, N=50, Not yet recruiting, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
New trial
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HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • HLA-B (Major Histocompatibility Complex, Class I, B) • HLA-C (Major Histocompatibility Complex, Class I, C)
|
cytarabine • fludarabine IV • thiotepa • busulfan