Chelation for iron overload in patients with PKD

Physicians monitor iron levels closely in order to adequately remove excess iron from the patient while at the same time preventing any potential drug toxicity. It should be noted that young children are more prone to iron chelator toxicity. The lower the LIC and SF the greater the chance of drug toxicity due to over-chelation.

Medications

Medications called iron chelators are the only option for iron removal in transfusion dependent patients and a commonly used method of removing iron in non-transfusion dependent patients with pyruvate kinase deficiency. Compliance with medications is of utmost importance for adequate iron unloading.  At this time there are three iron chelating medications available to patients.  For some patients, they may be used in combination.

They are:

1. Deferroxamine (Desferal)
2. Deferasirox (Exjade or Jadenu)
3. Deferiprone (L1)

Important note: Though these chelating medications have been found to be safe and effective in patients with thalassemia, because of the rarity of pk deficiency, there have been no comprehensive studies completed on large groups of patients with pkd using these chelators.

“Some nights I really, really want to skip wearing my Desferal pump, but then I picture the iron sitting in my organs…destroying them. That keeps me on track”
Female, PKD Patient

 Deferoxamine (Desferal)

Though Deferoxamine (Desferal, DFO) can be given intravenously, it is most often administered subcutaneously (with a short needle under the skin) using a portable infusion pump. Typically the pump is worn 8-15 hours, 5-7 days per week. Dosage and frequency is individualized and based on the degree of iron overload and the weight of the patient. DFO has been on the market and studied in patients longer than any other iron chelator available.

Click here for information on dosage, toxicity, and side effects of Desferal.

Deferasirox (Exjade)

Deferasirox (Exjade, Asunra, Desirox) is a daily oral chelator (tablet) that is mixed with water, orange juice, or apple juice. Although it is recommended that it be taken on an empty stomach about 30 minutes before or after eating, recent data indicate that taking Exjade with food is effective for patients who have difficulty taking it on an empty stomach. The most dangerous potential side effect of this medication is kidney damage. It is important that creatinine levels be monitored monthly. Gastrointestinal side effects (nausea, vomiting, diarrhea, abdominal pain) are the most common and usually subside over time. If these symptoms persist a doctor may reduce the dose or stop the drug and start it up again by gradually increasing the dose up to the desired level. Many patients have also responded with fewer side effects by splitting the prescribed dose into two – taking one half in the morning and one half in the evening.

Click here for more information on dosage, toxicity, and side effects of Exjade.

Deferasirox (Jadenu)

Jadenu is the newest iron chelator available. It is Deferasirox, but in a film coated tablet form that does not get dissolved. It is taken with water or another liquid once daily.

Click here for more information on dosage, toxicity, and side effects on Jadenu.

Deferiprone (Ferriprox)

Deferiprone (Ferriprox , L1, Kelfer) is an iron chelator that is administered to transfusion dependent patients, which is often picked as second or third choice  when their current chelator  is not effective or has intolerable side effects. It is a tablet that is taken orally 3 times per day. This drug can cause a very low white blood cell count (neutropenia). Severe neutropenia is called agranulocytosis, which can lead to significant illness or death. Therefore, any patient taking this drug needs to have his/her white blood cell counts checked once per week.

Click here for more information on dosage, toxicity, and side effects of Ferriprox.

References

Barton, James C., et al. Handbook of Iron Overload Disorders. 1^st ed. Cambridge: Cambridge University Press, 2010.

Vichinsky E, et al. Standards of Care Guidelines for Thalassemia. UCSF Benioff Children’s Hospital Oakland. 2000, revised 2009.

Aydinok Y, Kattamis A, Viprakasit V.  Current approach to iron chelation in children. Br J Haematol. 2014 Jun;165(6):745-55.

Taher AT, Viprakasit V, Musallam KM, Cappellini MD. Treating iron overload in patients with non-transfusion-dependent thalassemia. Am J Hematol 2013 May;88(5):409-15.

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