Elzonris(tagraxofusp-erzs)

Elzonris indications
For the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in children aged 2 years and older and in adult patients:

Treatment-naive patients
Previously treated patients
(Note: BPDCN is a rare and aggressive hematologic malignancy)

Elzonris specifications
Injection: 1,000 μg/1 mL (single-dose vial)
Elzonris mechanism of action
Antibody-drug conjugate (ADC) targeting CD123:

Targeting part: Interleukin-3 (IL-3) specifically binds to CD123 antigen on the surface of tumor cells
Cytotoxic part: Conjugated truncated diphtheria toxin causes tumor cell apoptosis
Unique mechanism: Selectively kills tumor cells that highly express CD123
Elzonris global approval status
U.S. FDA (approved on December 21, 2018):
The first BPDCM targeted therapeutic drug
Obtained orphan drug status and priority review
EU EMA (approved on January 7, 2021)
Elzonris usage and dosage
Standard dose:
12 μg/kg Intravenous infusion (each infusion time ≥15 minutes)
Dosing cycle: 1 cycle every 21 days, daily administration on days 1-5
Key management requirements:
Pretreatment medication: Administer 1 hour before infusion:
Corticosteroids (such as dexamethasone)
H1/H2 receptor antagonists
Antipyretic analgesics
Monitoring requirements:
Before treatment: test serum albumin, liver function, body weight
The first dose of each cycle: inpatient monitoring ≥24 hours
Subsequent infusions: outpatient monitoring ≥4 hours
Dose adjustment: Adverse reaction level treatment measures Capillary leak syndrome (CLS) Permanent discontinuation of drug Elevated liver enzymes (≥5 times ULN) Suspend medication until recovery
Elzonris adverse reactions
≥20% incidence (key research data):
Systemic reactions: fever (77%), peripheral edema (55%), fatigue (41%)
Digestive system: nausea (64%), vomiting (32%)
Laboratory abnormalities:
Hypoalbuminemia (83%)
Elevated liver enzymes (ALT 45%/AST 41%)
Black box warning:

Capillary leak syndrome (CLS):
Potentially life-threatening (manifested as hypotension, hypoalbuminemia, edema)
Weight needs to be monitored (increase ≥3% indicates risk)
Hepatotoxicity:
May progress to liver failure
Elzonris clinical research data
Key study STML-401-0114 (NCT02113982):

Patient type Number of cases ORR Median OS First-time patients 29 90% Not reached Experienced patients 15 67% 8.5 months

Survival data: First-time patients 24-month survival rate reaches 52%
Elzonris special population medication
Pediatric patients:
Safety of patients ≥2 years old is similar to that of adults
Data for patients <2 years old is missing Pregnancy: Animal studies show embryotoxicity (contraindicated during pregnancy) Lactation: Discontinue medication for ≥1 week after the end of lactation Elzonris drug interactions Hepatotoxic drugs (such as acetaminophen): increase the risk of liver damage Immunosuppressants: may increase the risk of infection Elzonris precautions CLS monitoring: Serum albumin needs to be ≥3.2 g/dL before treatment Monitor body weight daily (sudden increase of ≥3% needs to be vigilant) Infection prevention and control: Prophylactic use of antiviral/antibacterial drugs (if necessary) Infusion management: Intravenous infusion only (subcutaneous/intramuscular injection is prohibited) Dilution with 0.9% sodium chloride is required before infusion Elzonris FAQs Q: What should I do if chills occur after infusion? A: Immediately suspend the infusion and give symptomatic treatment (such as diphenhydramine). The infusion can be resumed after the symptoms are relieved Q: Can I get vaccinated during treatment? A: Live vaccines (such as MMR vaccine) are prohibited, and the response to inactivated vaccines may be reduced Q: How to judge the early symptoms of CLS? A: Pay attention to sudden weight gain, lower limb edema or dyspnea Q: Is routine prevention of CLS necessary? A: There is currently no standard prevention program, and strict monitoring is the key (Note: This manual integrates FDA/EMA approval information, please follow local regulations for specific use) Appendix: Important safety tips The first use must be carried out in a medical institution with rescue conditions The patient's liver function and cardiovascular status must be assessed before prescription