Deferasirox Instructions

(Deferasirox) Instructions

Generic name: Deferasirox

Product name: Enrig

All names: Deferasirox, Enrig, Deferasirox, Desirox

Indications:

It is used to treat chronic iron overload in patients with β-thalassemia older than 6 years old due to frequent blood transfusions (monthly administration of concentrated red blood cells ≥7mL/kg);

For children under 6 years old and iron overload caused by other transfusion-dependent diseases, the safety and effectiveness data of Enrig in Chinese patients are limited, and it is recommended to use it with caution based on the specific conditions of the patients. 

Usage and dosage:

Starting dose:

The recommended starting daily dose of deferasirox is 20 mg/kg.

For patients who receive monthly transfusions of more than 14 mL/kg of packed red blood cells (i.e., more than 4 units/month in adults) and who need to reduce excess iron exposure, a starting dose of 30 mg/kg/day may be considered.

For patients who receive monthly transfusions of less than 7 mL/kg of packed red blood cells (i.e., less than 2 units/month in adults) and who need to maintain body iron balance, a starting dose of 10 mg/kg/day may be considered.

In patients who have already responded well to deferoxamine therapy, an initial dose of Enriga equivalent to half the deferoxamine dose may be considered. (For example, a patient receiving deferoxamine 40 mg/kg/day, 5 days a week or equivalent, may start with 20 mg/kg if switched to Enriga.)

How to take:

Deferasirox should be taken once daily on an empty stomach, at least 30 minutes before a meal, preferably at the same time each day. Tablets should not be chewed or swallowed whole. 

Deferasirox should not be taken with aluminum-containing antacids, and the dose (mg/kg) needs to be calculated and rounded to the nearest whole tablet.

Completely dissolve the tablets in water, apple juice or orange juice (100-200mL) by stirring until a clear suspension is obtained and then drink it. The remaining medicine must be added with a small amount of water, apple juice or orange juice and mixed well before taking. Not recommended for dissolution in carbonated drinks or milk as it can cause foaming and slow dispersion. 

Adverse reactions:

>10%

Increased serum creatinine (dose-related; 7-38%)

Abdominal pain (21-28%)

Nausea (11-23%)

Vomiting (10-21%)

Diarrhea (12-20%)

Proteinuria (19%)

Getting angry (19%)

Headache (16%)

Cough (14%)

Nasopharyngitis (13%)

Sore throat (11%)

Flu (11%)

Rash (8-11%)

1-10%

Respiratory tract infection (10%)

Bronchitis (9%)

ALT increase (2-8%)

Joint pain, back pain (6-7%)

Acute tonsillitis (6%)

Rhinitis (6%)

Fatigue (6%)

Ear infection (5%)

Transaminases (4%)

Urticaria (4%)

<1%

allergic reaction

angioedema

Cytopenia, including agranulocytosis, neutropenia, and thrombocytopenia; leukocytoclastic vasculitis

Things to note:

allergies

Estimated GFR <40 ml/min/1.73m² or serum creatinine >2 x ULN

Poor performance status and high risk for myelodysplastic syndrome or advanced malignancy

Platelet count <50 x10^9/L

Storage:

Store at room temperature 25°C (77°F); excursions allowed 15-30°C (59-86°F)

Moisture proof

Mechanism of action:

DFS has pharmacological effects such as antifungal effects (such as Mucor mold that grows in iron-rich environments), anti-cell proliferation, anti-malarial effects, anti-oxidative stress damage, and anti-cytotoxicity-induced apoptosis.

1.DFS and fungal infection

Iron is a substance that fungi rely on for survival, and it plays a very important role in the growth and toxicity of fungi. The antifungal effect of iron chelators may lie in their removal of iron necessary for fungal growth, thereby achieving the purpose of treating fungal infections.

2.DFS and cell proliferation

Iron plays a very important role in the process of cell proliferation, and iron chelators may have anti-cell proliferation properties. This suggests that iron chelators may become a new type of anti-cancer agent for the treatment of tumors. 

Efficacy and safety:

The chemical name of (deferasirox) is 4-[3,5-bis(2-hydroxyphenyl)-1,2,4-triazol-1-yl]benzoic acid. It is an iron chelator product developed by the Swiss pharmaceutical company Novartis. It is the first oral iron-removing agent approved by the US FDA for routine use. It is approved for use in patients ≥2 years old with chronic iron overload caused by blood transfusion. In Europe, it is recommended as a 6 As a first-line drug for patients with thalassemia iron overload over the age of 18, clinical research is currently underway in China; Phase II and III clinical trials and pharmacokinetic studies have shown that it has good safety and tolerability, can significantly reduce iron load in the heart and liver, and is easily accepted by patients. At the same time, it also has pharmaceutical properties such as antifungal (such as Mucor that grows in an iron-rich environment), anti-cell proliferation, anti-malarial, anti-oxidative stress damage, anti-cytotoxic-induced apoptosis, etc.; it can be used for the treatment of secondary hemochromatosis, porphyria cutanea tarda and other diseases.