Monitoring and management of deferiprone adverse events

Adverse effect	Monitoring	Action
Hematologic Neutropenia^* Agranulocytosis (ANC <500/microL), most commonly occurs in the first several months of therapy	CBC with differential at baseline and weekly for the first 6 months of chelation therapy. For patients who have not had an interruption in therapy due to neutropenia, extend monitoring interval to every 2 weeks for 6 months, then every 2 to 4 weeks (or at the patient's transfusion interval) thereafter.	Avoid concomitant use of drugs that cause myelosuppression. Counsel patient to hold deferiprone and seek medical attention with all febrile illnesses. If ANC is <500/microL, discontinue therapy; due to high risk of recurrent agranulocytosis, do not restart therapy with deferiprone. If fever occurs with ANC <500/microL, admit to hospital, obtain blood cultures, and start broad spectrum antibiotics. For moderate neutropenia (ANC 500 to 1000/microL), hold deferiprone and recheck CBC every 2 to 3 days. Therapy may be resumed if ANC is >1500/microL. If ANC does not recover by 2 weeks, permanently discontinue deferiprone. For mild neutropenia (ANC 1000 to 1500/microL) deferiprone may be continued with close monitoring; monitor ANC every 2 to 3 days until recovery and hold deferiprone if the ANC falls below 1000/microL.
Liver Transient transaminase elevations^¶	Obtain ALT and bilirubin monthly.	If normal at baseline and ALT increases to >3 times the upper limit of normal on 2 consecutive tests, lower the dose by 25%. If normal at baseline and ALT increases >10 times the upper limit of normal or direct bilirubin increases >2 times the upper limit of normal, hold therapy and restart at a lower dose (50% of the prior dose) once ALT and direct bilirubin normalize. Monitor transaminases and bilirubin, and gradually increase the dose if tolerated. If abnormalities recur, consider switching to a different chelator. If abnormal at baseline and increases >2 times baseline and >5 times the upper limit of normal in patient with very elevated liver iron concentration or chronic viral hepatitis, lower dose by 50%; if no improvement in ALT after 1 month, hold the dose. If no improvement in ALT after holding deferiprone for 1 month, may resume at higher dose (eg, 25% increase) with close monitoring.^¶
Bone Arthralgias	Obtain history and perform physical examination at every visit.	Mild arthralgias may be treated with an NSAID, if no contraindications. Hold deferiprone for severe arthralgias. Deferiprone may be restarted at a lower dose (25 to 50% lower) once symptoms resolve; dose may be increased if tolerated. Consider imaging (radiography, MRI) for persistent or severe symptoms.
Zinc deficiency	Zinc level annually.	Supplement if low.

Deferiprone (Ferriprox)-induced neutropenia is most common during the first year of therapy but can occur at any time. Refer to UpToDate for details of chelator selection, dosing, and adverse effects.

ALT: alanine aminotransferase; ANC: absolute neutrophil count; AST: aspartate aminotransferase; CBC: complete blood count; MRI: magnetic resonance imaging; NSAID: nonsteroidal antiinflammatory drug.

* ANC <500/microL is considered agranulocytosis; ANC 500 to 1000/microL is considered moderate neutropenia; ANC 1000 to 1500/microL is considered mild neutropenia. An ANC calculator is available within UpToDate.

¶ Increases in ALT and AST occur in up to 30% of patients on initiation of deferiprone and often resolve spontaneously without drug discontinuation. If the elevation persists despite discontinuation of deferiprone, it is presumed not to be due to the drug.

References:

Ferriprox (deferiprone) tablets. United States prescribing information. Revised March 2025. US Food and Drug Administration. https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/021825s012lbl.pdf (Accessed on May 9, 2025).
Ware HM, Kwiatkowski JL. Evaluation and treatment of transfusional iron overload in children. Pediatr Clin North Am 2013; 60;1393.

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Monitoring and management of deferiprone adverse events

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