Among the 59 pediatric patients, 34 cases (58%) were treated with trimethoprim-sulfamethoxazole monotherapy, 19 cases (32%) received combination therapy with micafungin, and 7 cases (12%) received combination therapy with clindamycin. Clinical manifestations and imaging findings lack specificity, and mNGS facilitates early diagnosis. The core treatment is trimethoprim-sulfamethoxazole, most children require respiratory support, and combination with low-dose glucocorticoids may improve prognosis.

P=N/A, N=260, Recruiting, University Hospital, Strasbourg, France

P3, N=1192, Not yet recruiting, Beijing Tiantan Hospital

P1, N=8, Terminated, University of Oxford | N=16 --> 8 | Trial completion date: Aug 2026 --> Apr 2025 | Recruiting --> Terminated | Trial primary completion date: Aug 2026 --> Apr 2025; The decision was made due to the expired investigational drug and regulatory changes by the Thai FDA, along with increased importation costs. The research team and investigational drug sponsor agreed to seek approval for early project termination.

Through a high-throughput screen of FDA-approved compounds, artemether (ART) emerged as a robust inducer of PD-L1 surface expression in multiple murine and human TNBC cell lines. Critically, in syngeneic TNBC mouse models, ART synergistically enhanced the antitumor efficacy of atezolizumab, promoting tumor regression and increasing intratumoral CD8+ T cell infiltration and cytotoxicity. Our findings reveal a novel mechanism by which ART upregulates PD-L1 through KEAP1 inhibition and establish ART as a promising pharmacological agent to improve the clinical outcomes of PD-L1 checkpoint blockade immunotherapy in TNBC.

P2 exhibited a ∼10-fold increase in antiproliferative potency against human leukemic cell lines compared to homoharringtonine (HHT)...Our findings provide valuable insights to guide the future structural optimization of harringtonine derivatives. Furthermore, P2 has been identified as a promising anti-leukemic candidate and warrants further development.

Moreover, thiostrepton treatment sensitized the CTCL cells to proteasome inhibitor bortezomib, promoting apoptosis and autophagy. Collectively, these findings demonstrate that FOXM1 targeting disrupts the metabolic status and stemness features of CTCL cells via JNK activation, thereby offering novel insights into potential therapeutic strategies for overcoming therapeutic challenges in CTCL.

P=N/A, N=210, Active, not recruiting, Psychiatric University Hospital, Zurich | Recruiting --> Active, not recruiting

Oral tetracyclines appear to reduce the incidence of all-grade acneiform rash or, alternatively, to decrease the incidence of high-grade rash. Preventive treatment for acneiform rash at the initiation of epidermal growth factor receptor tyrosine kinase inhibitor therapy should therefore be considered. Further controlled trials are needed to confirm the efficacy of oral tetracyclines in preventing acneiform rash.

P=N/A, N=100, Recruiting, Western Galilee Hospital-Nahariya

In vivo, the anti-leukemic efficacy of HHT was significantly diminished upon EWSR1 knockdown, demonstrating that EWSR1 was required for therapeutic response. Collectively, these findings uncover a phase separation-centric mechanism by which HHT exerts anti-AML activity, establish the EWSR1-YTHDF2-m6A axis as a critical regulator of leukemia progression, and position EWSR1 as both a functional target and a predictive biomarker for optimizing HHT-based therapies.

This study highlights the value of computational drug repurposing platforms for accelerating therapeutic discovery. Further preclinical investigations are warranted to evaluate HHT's in vivo efficacy and clinical applicability in RPF.