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Answered Questions...
- In compliant patients who do not achieve a satisfactory response to DFO, what factors would suggest moving to a combination of DFO and DFP and what factors would prompt you to switch to DFX?
Reveal answer
Although the combination of DFO (twice weekly SC) plus DFP (three times/day orally) significantly increases the probability of achieving a response, this regimen may be difficult for some patients to adhere to. Advantages offered by DFX are the efficacy of this agent and the convenience of a once-daily oral regimen, particularly in patients who are having difficulty with SC administration. Of course, the side effect profiles of the two regimens also impact the choice of therapy. Neutropenia and, less frequently, agranulocytosis, have been observed with DFO + DFP, whereas side effects most commonly associated with DFX are gastrointestinal disturbances and rash.
- What is the first step you would take in assessing non-response?
Reveal answer
Since the primary reason for drug failure with any regimen is the patient's failure to take the drug, the first thing is to ascertain the patient's adherence to therapy. Following that, you should determine the patient's transfusion loading rate, try an alternate measure of response, and see if there are other issues, such an inadequate dose requiring dose titration, comorbid diseases that may confound the assessment of response, or the timing of oral dosing with food intake, etc, that may be a factor.
- Are there similar issues with bioavailability of DFO and DFP that have been observed with DFX?
Reveal answer
It's important to note that data on interpatient differences in bioavailability are scanty even in terms of DFX, and exactly what accounts for patient differences in bioavailability of DFX is unclear. With the exception of oral absorption, which is not an issue with an agent administered SC, patient differences in distribution, metabolism, and excretion could exist with all chelators. At this point, we don't know.
- Do patients take advantage of peer support groups/family groups in your experience?
Reveal answer
As mentioned, these groups are part of a larger program to improve adherence that includes disease education, medication diaries, and positive reinforcement from the staff for good compliance. Patients and their families derive considerable benefit from the support groups and the program has had a positive impact. But compliance still remains a problem.
- Should the initial starting dose of chelation be based on a combined calculation of transfusion rate and body iron stores or should one start at the recommended initial dose and adjust accordingly depending on response?
Reveal answer
This issue was only investigated for the oral chelator deferasirox. The current recommendation is a fixed starting dose based on iron intake from ongoing blood transfusions and current iron burden with subsequent individual dose titration every 3 months according to serum ferritin levels and safety markers. One important question to answer during dose assignment is whether the target is to reduce or maintain body iron levels.
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