Promacta (eltrombopag)

P2, N=80, Recruiting, National Heart, Lung, and Blood Institute (NHLBI) | N=39 --> 80

P2, N=34, Active, not recruiting, National Heart, Lung, and Blood Institute (NHLBI) | Trial completion date: Mar 2025 --> Jun 2025

P1/2, N=15, Completed, National Heart, Lung, and Blood Institute (NHLBI) | Active, not recruiting --> Completed

P2, N=35, Active, not recruiting, Novartis Pharmaceuticals | Recruiting --> Active, not recruiting

Consequently, we determined that the patient presented with a condition similar to immune thrombocytopenic purpura (ITP) and selected a treatment approach consisting of eltrombopag, a thrombopoietin receptor agonist. Although uncommon, anti-HER2 antibodies can cause severe thrombocytopenia. Furthermore, if thrombocytopenia persists after treatment discontinuation and the administration of corticosteroids, exploring treatment options aligned with managing ITP is essential.

P2, N=110, Active, not recruiting, French Innovative Leukemia Organisation | Trial completion date: Sep 2024 --> Jul 2026

P2, N=2, Completed, University Children's Hospital Basel | Active, not recruiting --> Completed | Trial completion date: Oct 2024 --> Apr 2024 | Trial primary completion date: Oct 2024 --> Apr 2024

P2, N=30, Recruiting, M.D. Anderson Cancer Center | Not yet recruiting --> Recruiting

P2, N=25, Not yet recruiting, Case Comprehensive Cancer Center

P1/2, N=20, Not yet recruiting, Melbourne Health

P2, N=13, Completed, University Hospital, Toulouse | Recruiting --> Completed | N=25 --> 13

SCC of the head and neck or gynecologic organs in a young adult without known risk factors should prompt consideration of FA. Cisplatin should be avoided in patients with FA.

P2, N=36, Recruiting, Novartis Pharmaceuticals | Trial completion date: Aug 2027 --> Feb 2027 | Trial primary completion date: Jan 2026 --> Jul 2025

P4, N=157, Completed, Institute of Hematology & Blood Diseases Hospital, China | Active, not recruiting --> Completed

P=N/A, N=10, Recruiting, Novartis Pharmaceuticals | Not yet recruiting --> Recruiting

Our aim is to determine differentially expressed genes (DEGs) related to Asparaginase, Daunorubicin, Prednisolone, and Vincristine resistance and identify potential inhibitors via docking...Following ADMET analysis, three drugs emerged as potential inhibitors: Flunarizine, Talniflumate, and Eltrombopag...This study promotes a further understanding of drug resistance in ALL, introducing novel genes for consideration in diagnostic screening. It also presents potential inhibitor candidates to tackle drug resistance through repurposing.

P3, N=3, Not yet recruiting, Assiut University

BCAA deprivation alone did not effectively inhibit PCC proliferation. However, BCAA deprivation combined with eltrombopag, a drug targeting TFEB, can play a two-pronged role in exogenous supply deprivation and endogenous utilisation blockade to inhibit the proliferation of pancreatic cancer to the greatest extent, providing a new therapeutic direction, such as targeted metabolic reprogramming of pancreatic cancer.

P=N/A, N=126, Recruiting, Yunfeng Cheng

P=N/A, N=10, Not yet recruiting, Novartis Pharmaceuticals | Trial completion date: Oct 2028 --> Mar 2029 | Trial primary completion date: Oct 2028 --> Mar 2029

P=N/A, N=52, Completed, Tongji Hospital

Chimeric antigen receptor T (CAR-T) cell therapy (lisocabtagene maraleucel; liso-cel) has been used to treat a few cases of isolated secondary CNS lymphoma. Herein, we report the case of a 66-year-old male diagnosed with diffuse large B-cell lymphoma (Ann Arbor grade IV; R-IPI, good risk; CNS IPI: Intermediate risk) who achieved complete remission (CR) after six courses of R-CHOP therapy...Seven high-dose methotrexate courses led to partial response. Subsequently, the patient received CAR-T cell therapy with tolerable adverse events - cytokine release syndrome treated with tocilizumab, no immune effector cell-associated neurotoxicity syndrome, and bone marrow failure treated with granulocyte-colony stimulating factor and eltrombopag...This case is the first to validate the efficacy and safety of CAR-T cell therapy for isolated secondary CNS lymphoma in clinical practice. Future accumulation of evidence on the efficacy and safety of CAR-T cell therapy is essential.

P3, N=150, Recruiting, Novartis Pharmaceuticals | Trial completion date: Feb 2028 --> May 2028

The growth inhibitory effect of these final shortlisted compounds was examined on a panel of GBM cell lines and compared with temozolomide through the drug sensitivity EC50 values and AUC from the PRISM Repurposing Secondary Screen, and the IC50 values were obtained from GDSC portal...Our results show GSK-2126458/omipalisib, linifanib, drospirenone, eltrombopag, nilotinib, and PD198306 as candidate drugs which can be further evaluated for their anti-tumor potential against GBM. Through this work, we identified repurposed candidate therapeutics against GBM utilizing a GSC inclusive targeting approach, which demonstrated high in vitro efficacy and can prospectively evade drug resistance. These drugs have the potential to be developed as individual or combination therapy to improve GBM outcomes.

P2, N=30, Not yet recruiting, M.D. Anderson Cancer Center

P2, N=132, Terminated, Technische Universität Dresden | N=238 --> 132

P3, N=122, Active, not recruiting, Baylor College of Medicine | Recruiting --> Active, not recruiting

P2, N=39, Recruiting, National Heart, Lung, and Blood Institute (NHLBI) | Trial primary completion date: Mar 2024 --> Mar 2025

P1, N=16, Completed, Mirati Therapeutics Inc. | Recruiting --> Completed | Trial completion date: Aug 2024 --> Aug 2023

P=N/A, N=10, Not yet recruiting, Novartis Pharmaceuticals

P4, N=157, Active, not recruiting, Institute of Hematology & Blood Diseases Hospital, China | Recruiting --> Active, not recruiting | Trial completion date: Dec 2024 --> Jul 2024 | Trial primary completion date: Jun 2024 --> Feb 2024

P2, N=10, Terminated, Novartis Pharmaceuticals | Completed --> Terminated; In the context of COVID-19 pandemic, the benefit / risk ratio of the participation of a patient with pancytopenia could be compromised. The decision of this premature termination was not the consequence of any safety reason inherent to the drug.

P2, N=28, Completed, Novartis Pharmaceuticals | Active, not recruiting --> Completed

P4, N=60, Completed, Sobi, Inc. | Active, not recruiting --> Completed

P1, N=20, Active, not recruiting, University of California, San Francisco | Trial completion date: Dec 2023 --> Dec 2024 | Trial primary completion date: Dec 2023 --> Dec 2024

P2, N=2, Active, not recruiting, University Children's Hospital Basel | Recruiting --> Active, not recruiting | N=24 --> 2

P=N/A, N=100, Not yet recruiting, Assiut University

Thrombopoietin agonists such as eltrombopag (EPAG) or CD34-selected cell infusions are treatments offered for persistent cytopenias...Patient C1 who showed a spontaneous recovery of counts without the need of EPAG had the highest proportion of LT-HSCs despite lying in the second quartile of the cohort for both B cells as well as B-LPs. In conclusion, MP-FCM based analysis of the HSPC landscape in PGF marrow could be a robust prognostic biomarker in Allo-SCT patients.

Case Presentation: We present a 14-year-old Hispanic male with Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL) who received a maternal haploidentical HSCT with myeloablative conditioning and post-transplant cyclophosphamide...Multiple treatments including romiplostim; eltrombopag; high dose immune globulin (IVIG); rituximab; methylprednisolone; and decitabine were not successful in improving platelet count (Figure 1)...Patient continues to maintain platelets above 100 x10 3/uL with no medication toxicities or adverse events noted. Patient has been able to return to his pre-alloHSCT lifestyle including contact sports and re-immunizations.

For patients who previously received corticosteroids and a TPO-RA, the choice of subsequent therapy (eg a different TPO-RA, rituximab, fostamatinib, mycophenolate mofetil, splenectomy) is not well defined. VAYHIT3 will evaluate the ability of a short therapeutic course of ianalumab to induce durable responses in patients who have received 2 or more prior lines of therapy, including at least 1 corticosteroid and 1 TPO-RA, for whom significant unmet needs exist. Current status: VAYHIT3 is recruiting. Ianalumab is also being investigated in 2 ongoing, randomized, double-blind, Phase III ITP trials: VAYHIT1 (NCT05653349) is assessing ianalumab versus placebo in addition to first-line corticosteroids, and VAYHIT2 (NCT05653219) is assessing ianalumab versus placebo in addition to eltrombopag in patients who failed first-line corticosteroid treatment.

All family members have normal blood values and none harbored the mutation. Here, we will discuss the unusual evolution of this case and revisit the literature.

P3, N=96, Recruiting, Peking University People's Hospital | Not yet recruiting --> Recruiting | Phase classification: P2 --> P3