cladribine

P2, N=100, Recruiting, M.D. Anderson Cancer Center | Trial completion date: Dec 2025 --> Dec 2027 | Trial primary completion date: Dec 2025 --> Dec 2027

Treatments included vemurafenib, interferon, tocilizumab, cladribine, cobimetinib, and cytarabine. Treatment responses varied, with several patients achieving remission or stable disease, while others had progressive or end-stage disease. This study highlights the clinical heterogeneity of ECD and the value of integrating histopathology, molecular profiling, and imaging to guide management and improve outcomes.

P1/2, N=62, Recruiting, University of Washington | Trial completion date: Dec 2026 --> Dec 2027

P1/2, N=84, Recruiting, National Cancer Institute (NCI) | Not yet recruiting --> Recruiting | Initiation date: Sep 2025 --> Jun 2026

P2, N=68, Recruiting, M.D. Anderson Cancer Center | Not yet recruiting --> Recruiting

P2, N=116, Active, not recruiting, M.D. Anderson Cancer Center | Trial completion date: Oct 2025 --> Oct 2027 | Trial primary completion date: Oct 2025 --> Oct 2027

P2/3, N=324, Recruiting, M.D. Anderson Cancer Center | Phase classification: P2 --> P2/3

The patient was treated with cladribine and rituximab and achieved hematological recovery. This case highlights the usefulness of FCM for the diagnosis of HCL in the presence of splenomegaly, pancytopenia, and myelofibrosis, even though hairy cells may be absent in peripheral blood.

Hairy cell leukemia (HCL) is a rare, indolent B-cell lymphoma that is highly responsive to the purine analog cladribine...Further research is needed to better understand the pathophysiological links between HCL, large cell transformation, and HLH. Additionally, registry or other cross-sectional studies with larger numbers of HCL patients could clarify the exact frequency of these rare but fatal complications.

Somatic mutations, gene expression, and functional drug testing are important for the selection of small molecule inhibitors of oncogenic signalling pathways (e.g., FLT3), menin inhibitors that disrupt leukemogenic programmes, inhibitors of isocitrate dehydrogenases to restore oncometabolic homoeostasis, and proapoptotic Bcl-2 homology 3 (BH3) mimetics, such as venetoclax. Targeting the recently identified resistance factor SAMHD1 promises to overcome resistance to cytarabine and fludarabine. Given the growing number of potential combinatorial drug regimens and the genetic heterogeneity of AML, real-time ex vivo drug response profiling to guide individualized treatment decisions will become an important complement. We argue that better outcomes for R/R AML critically depend on being guided by precision oncology to define the best combination of chemotherapy, targeted therapy, and immunological therapy informed by phenotypic and genotypic patient- and disease-specific parameters.

P1, N=0, Withdrawn, M.D. Anderson Cancer Center | N=22 --> 0 | Trial completion date: Dec 2033 --> Jul 2025 | Not yet recruiting --> Withdrawn | Trial primary completion date: Dec 2031 --> Jul 2025