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DRUG CLASS:

DNA-directed DNAP inhibitor

4d
Gilteritinib + chemotherapy in children with relapsed/refractory FLT3-ITD AML: results from the phase 1/2 SKIPPER trial. (PubMed, Blood Adv)
The multi-national phase 1/2 SKIPPER study evaluated gilteritinib combined with fludarabine and cytarabine chemotherapy and granulocyte colony‑stimulating factor (FLAG) in children and adolescents/young adults with relapsed/refractory FLT3‑ITD AML. The gilteritinib and FLAG regimen had manageable safety, induced high remission rates, and enabled allogeneic hematopoietic stem cell transplant for several patients. Although limited by a small sample size, these findings support further evaluation of gilteritinib in pediatric patients with FLT3-mutated AML, particularly in frontline settings (ClinicalTrials.gov Identifier: NCT04240002).
P1/2 data • Journal
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FLT3 (Fms-related tyrosine kinase 3)
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FLT3 mutation
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cytarabine • Xospata (gilteritinib) • fludarabine IV
6d
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
8d
Hepatitis B: A Review. (PubMed, JAMA)
Antiviral therapies-pegylated interferon alfa and nucleos(t)ide analogues (entecavir or tenofovir)-suppress HBV DNA replication and reduce the risk of HCC by approximately 50%...Universal HBV vaccination, particularly birth-dose administration, is the most effective strategy to prevent HBV infection. Among patients with HBV infection, antiviral therapy decreases progression to cirrhosis and liver failure and reduces the risk of HCC.
Journal
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AFP (Alpha-fetoprotein)
10d
Intravenous Brincidofovir as an Antiviral for Treatment of Progressive Multifocal Leukoencephalopathy: A Pilot Study (clinicaltrials.gov)
P2, N=24, Recruiting, National Institute of Neurological Disorders and Stroke (NINDS) | Not yet recruiting --> Recruiting
Enrollment open
12d
Multi-omics analysis of pediatric minimally differentiated acute myeloid leukemia reveals RUNX1-driven stemness and chemoresistance. (PubMed, Leukemia)
RUNX1 disruption was also associated with reduced in vitro sensitivity to multiple drugs, including cytarabine and anthracyclines. Our study provides the most comprehensive molecular characterization of pediatric AML-M0 to date and identifies RUNX1 alterations as important biological and clinical determinants. These insights highlight the potential strategies for precision therapy, including hypomethylating agents, signaling inhibitors, and metabolic targeting, to improve outcomes.
Journal
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FLT3 (Fms-related tyrosine kinase 3) • RUNX1 (RUNX Family Transcription Factor 1) • ETV6 (ETS Variant Transcription Factor 6)
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cytarabine
13d
A Rare Extramedullary Presentation of Acute Myeloid Leukemia: Myeloid Sarcoma Presenting as a Hard Scrotal Swelling. (PubMed, Cureus)
The patient received induction chemotherapy with cytarabine and daunorubicin along with localized radiotherapy. This case highlights the importance of considering hematologic malignancies in patients presenting with atypical or treatment-refractory scrotal swellings. Early biopsy and multidisciplinary evaluation are essential for timely diagnosis and appropriate management.
Journal
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CD34 (CD34 molecule) • MPO (Myeloperoxidase)
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cytarabine • daunorubicin
18d
Roles of RRM2 and RRM2B in pyrimidine stress responses and differentiation of acute myeloid leukemia cells. (PubMed, Cell Death Discov)
Although brequinar and cytarabine altered nucleotide metabolism through distinct mechanisms, differentiation induced by all agents was abolished by supplementation with high levels of ribo- and deoxyribonucleosides, confirming that nucleotide imbalance is a central driver. Consistent with these differences, RRM2 depletion enhanced differentiation in U937 cells without affecting viability but impaired differentiation and survival in MOLM-13 cells. These findings position nucleotide metabolism as a key regulator of AML differentiation and suggest that combining RNR-targeted and checkpoint-modulating strategies could optimize therapeutic responses.
Journal
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RRM2 (Ribonucleotide Reductase Regulatory Subunit M2)
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TP53 mutation • TP53 wild-type
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cytarabine • brequinar (DUP 785)
18d
Diffuse Alveolar Hemorrhage as the Initial Manifestation of Acute Myelomonocytic Leukemia. (PubMed, Case Rep Hematol)
The patient was treated with induction chemotherapy (7 + 3 regimen: daunorubicin and cytarabine), but her condition deteriorated, and she eventually succumbed to respiratory failure. This case highlights the importance of considering hematologic malignancy in the differential diagnosis of DAH, particularly in the absence of other identifiable causes.
Journal
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FLT3 (Fms-related tyrosine kinase 3)
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FLT3 mutation
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cytarabine • daunorubicin
18d
A case report of multifocal gastrointestinal myeloid sarcoma without leukemic involvement: an unusual presentation posing a diagnostic challenge. (PubMed, Leuk Res Rep)
The patient was treated with standard induction chemotherapy using the "7 + 3″ regimen consisting of cytarabine and daunorubicin. During treatment, she developed fever, jaundice, and profound pancytopenia, and on day 12 she suffered sudden cardiorespiratory arrest and died. This case underscores the diagnostic complexity of gastrointestinal myeloid sarcoma, highlights the importance of immunohistochemistry in distinguishing it from other malignancies, and emphasizes the aggressive clinical course and poor prognosis that may occur even in non-leukemic presentations.
Journal • IO biomarker
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BCL2 (B-cell CLL/lymphoma 2) • NCAM1 (Neural cell adhesion molecule 1) • SPN (Sialophorin) • MPO (Myeloperoxidase)
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cytarabine • daunorubicin
18d
Efficacy of Ganciclovir Combined With Recombinant Human Interferon α-1b for Viral Encephalitis in Children (PubMed, Sichuan Da Xue Xue Bao Yi Xue Ban)
There was no significant difference in the total incidence rate of adverse reactions between groups (P > 0.05). Ganciclovir combined with recombinant human interferon α-1b can effectively treat viral encephalitis in children, improve cerebral hemodynamics and neurological function, enhance immune function, and relieve inflammatory response, and it dose not increase adverse reactions.
Clinical • Journal
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TNFA (Tumor Necrosis Factor-Alpha) • IL2 (Interleukin 2) • IL1B (Interleukin 1, beta)
19d
New trial • Head-to-Head
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Valcyte (valganciclovir)
19d
Efficacy and Safety of Brivudine in the Treatment of Herpes Zoster (ChiCTR2600118166)
P=N/A, N=140, Peking University Third Hospital; Peking University Third Hospital
New trial