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DRUG CLASS:

Purinergic receptor P1 agonist

2ms
Hidden in Plain Sight: Systemic Mastocytosis Manifesting as Isolated Hepatosplenomegaly in the Absence of Cutaneous and Classical Manifestations-A Case Report and Literature Review. (PubMed, Clin Case Rep)
The patient was treated with cladribine (40 mg over five days) followed by maintenance hydroxyurea (300 mg twice daily), with significant clinical improvement at eight-week follow-up. This case underscores that SM-AHN can present with isolated hepatosplenomegaly and profound leukocytosis without cutaneous signs, and highlights the critical role of integrated molecular profiling, including KIT mutation analysis, in the diagnostic workup of atypical hematologic presentations.
Journal
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ABL1 (ABL proto-oncogene 1) • BCR (BCR Activator Of RhoGEF And GTPase) • KIT (KIT proto-oncogene, receptor tyrosine kinase)
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cladribine • hydroxyurea
2ms
From Marginal to Central: Marginal Zone-like B Cells as Critical Targets in Cladribine-Treated Multiple Sclerosis. (PubMed, Ann Neurol)
MZ-like B cells are a dominant, polyfunctional inflammatory subset in MS, equipped for non-canonical lipid antigen presentation and IgM secretion. They represent a preferential pharmacological target of cladribine. Sustained reduction of this pathogenic memory pool, followed by repopulation with less inflammatory B cells, offers a mechanistic basis for cladribine's durable clinical benefit and nominates MZ-like cells as biomarkers of response and therapeutic targets. ANN NEUROL 2026.
Journal
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IL6 (Interleukin 6) • TNFA (Tumor Necrosis Factor-Alpha) • CSF2 (Colony stimulating factor 2) • CD1C (CD1c Molecule) • CD1D (CD1d Molecule)
2ms
Weekly Cladribine Followed by Rituximab for the Treatment of Hairy Cell Leukemia. (PubMed, EJHaem)
However, interpretation of these findings is limited by the non-randomized design and differences in rituximab exposure between groups, which may have contributed to the observed outcomes and limited definitive conclusions. Trial Registration: The authors have confirmed clinical trial registration is not needed for this submission.
Journal
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BRAF (B-raf proto-oncogene)
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BRAF V600E • BRAF V600
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Rituxan (rituximab) • cladribine
3ms
Hairy Cell Leukemia: Clinical Characteristics and Outcomes from a Single Center in the Middle East and North Africa. (PubMed, J Blood Med)
Treatment regimens included Cladribine alone or with Rituximab, yielding high response rates and minimal toxicity. In this multiethnic Middle Eastern and North African cohort, HCL predominantly affected middle-aged men and was often incidentally detected. Cladribine-based therapy achieved durable remissions with excellent survival, highlighting consistent efficacy across diverse populations.
Journal
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BRAF (B-raf proto-oncogene) • PAX5 (Paired Box 5) • ITGAE (Integrin Subunit Alpha E)
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BRAF V600E • BRAF V600
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Rituxan (rituximab) • cladribine
3ms
DUSP1 is a Key Driver of Disease Persistence and Potential Therapeutic Target in Hairy Cell Leukemia. (PubMed, Blood Adv)
Standard treatment with purine analogs (i.e. cladribine) induces long-term remissions, but up to 25% of patients relapse early...The functional importance of DUSP1 was corroborated by demonstrating that BMSC-induced protection from cell death could be overcome through DUSP1 inhibition. Our results might set the stage for future clinical testing of DUSP1 inhibition to eliminate minimal residual disease (MRD) and prevent disease relapse in HCL.
Journal
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DUSP1 (Dual Specificity Phosphatase 1)
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BRAF V600E • BRAF V600
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cladribine
3ms
Efficacy and Safety of a New Formulation of Oral Cladribine Compared With Placebo in Participants With Generalized Myasthenia Gravis (MyClad) (clinicaltrials.gov)
P3, N=264, Recruiting, Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany | Trial primary completion date: May 2028 --> Aug 2028
Trial primary completion date
3ms
Langerhans Cell Histiocytosis Associated With Chronic Myeloid Leukemia: A Pediatric Case Report. (PubMed, Cancer Rep (Hoboken))
This rare association of CML with LCH expands the recognized spectrum of LCH-associated malignancies. It underscores the importance of long-term surveillance in LCH patients, with particular vigilance for secondary malignancies. It also highlights the need for further research into possible biological relationships between histiocytic and myeloid neoplasms.
Journal
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ABL1 (ABL proto-oncogene 1)
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imatinib • cladribine • vinblastine
3ms
Prognostic impact of ASXL1 mutations in acute myeloid leukemia treated with lower intensity therapy. (PubMed, Cancer)
Collectively, these findings suggest that ASXL1MUT AML may be more appropriately classified as intermediate risk in the context of LIT+VEN-based therapy, with the depth of impact influenced by the specific LIT backbone.
Retrospective data • Journal
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TP53 (Tumor protein P53) • FLT3 (Fms-related tyrosine kinase 3) • ASXL1 (ASXL Transcriptional Regulator 1)
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TP53 mutation • FLT3-ITD mutation • RAS mutation • ASXL1 mutation
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Venclexta (venetoclax) • cytarabine • cladribine
4ms
Enrollment open
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cladribine • fludarabine IV • busulfan
4ms
Successful treatment of hairy cell leukemia with TP53 abnormality using cladribine combined with low-dose rituximab: a case report and literature review. (PubMed, Anticancer Drugs)
This case suggests that the combination of cladribine and low-dose rituximab may be an effective therapeutic strategy for classical HCL with TP53 abnormality, enabling deep molecular response and reversal of associated bone marrow fibrosis. Further prospective studies are warranted to validate these findings.
Journal
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BRAF (B-raf proto-oncogene) • TP53 (Tumor protein P53)
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TP53 mutation • BRAF V600E • BRAF V600 • TP53 deletion
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Rituxan (rituximab) • cladribine
4ms
New P4 trial
4ms
A Phase 1, Open-Label, Sequential Cross-over, Bioavailability/Bioequivalence Study to Compare the Pharmacokinetics of Oral Cladribine With the Reference Listed Drug, Intravenous Cladribine (clinicaltrials.gov)
P1, N=3, Terminated, M.D. Anderson Cancer Center | N=40 --> 3 | Trial completion date: Dec 2027 --> Mar 2026 | Active, not recruiting --> Terminated | Trial primary completion date: Dec 2027 --> Mar 2026; Slow participant enrollment
Enrollment change • Trial completion date • Trial termination • Trial primary completion date