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Recently, both of my new transplant consults were patients with chronic myeloid leukemia, a disease not often seen in transplant clinics anymore. In many ways, chronic myeloid leukemia is an ideal disease for stem cell transplantation. Outcomes are very good — with a more than 80% long-term survival in some series — and this may relate to the robust graft vs. tumor effect in this condition. However, with the advent of imatinib, physicians and patients came to view a lifetime of treatment with a tyrosine kinase inhibitor as preferable to the upfront risks for death and disability with transplantation.
Every once in a while, patients with CML still meet accepted indications for transplant. Patients may develop resistance to all of the available TKIs or become unable to tolerate these drugs.
And so it was that I met a young woman in her 20s on a second-line TKI (nilotinib) after a suboptimal response to imatinib. She was compliant and said she took all of her prescribed doses, and I had no reason to doubt her.
She had presented with fatigue, sweats and a very high white blood count about a year ago. Peripheral blood and marrow testing showed chronic phase CML and an unusual amount of marrow fibrosis. Her initial response to imatinib was as expected, with an early complete hematologic response. Fluorescence in situ hybridization (FISH) positivity of the peripheral blood at 9 months remained higher than hoped for, however, and BCR-ABL transcripts failed to decrease between 6 and 9 months.
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Medication switch
After data regarding the efficacy of dasatinib and imatinib in front-line CML treatment were published in The New England Journal of Medicine, her treating physicians changed her medication to nilotinib 400 mg twice daily to procure a better response. They chose this particular nilotinib dose after classifying her disease as resistant to imatinib.
Three months into treatment with nilotinib, repeat bone marrow and peripheral blood FISH testing were performed and results did not appear substantially different than the prior values. The BCR-ABL transcript level was only marginally improved. On this basis, because of concerns of nilotinib resistance and the likely need for a third-line TKI, the patient was referred to see me to discuss the role of eventual allogeneic stem cell transplantation.
Patient questionnaire
We ask all of our new patients in the transplant clinic to fill out a questionnaire that solicits sociodemographic data, past medical history and prior treatment details. As I was reviewing the entirety of the questionnaire in my office, my eye was drawn to the medication section, in which the patient had listed her nilotinib dosage as “200 mg twice daily.” I wasn’t expecting to see this, as the online medical record had faithfully recorded double this dosage for the prior 3 months.
I mentioned this to the treating physician, who subsequently contacted the patient and found that there had been an original misunderstanding about the intended dose. With each clinic visit, when asked whether she was taking every dose of her nilotinib as prescribed, the patient had promised that she was. She just hadn’t realized that she was to take two capsules each time instead of one. After that phone conversation, though, she began to take 400 mg twice daily, rather than 200 mg twice daily.
In the end, I don’t know whether this patient will get a much better response to nilotinib 400 mg twice daily than she did to 200 mg twice daily. It would be ironic if the most important intervention in the transplant consultation that day was having the patient provide her own information about her medication dosage, rather than relying on the truthfulness of the online medical record.
I’m looking forward to seeing how this patient does with her higher-dose nilotinib. If she improves, this experience will serve as a valuable reminder for me that it is what our patients say, and not what we as their clinicians think, that really matters after all.
William Wood, MD, is assistant professor of medicine, division of hematology/oncology, at the University of North Carolina Chapel Hill. Dr. Wood may be reached at william_wood@med.unc.edu.
For more information:
- Kantarjian H. N Engl J Med. 2010;362:2260-2270.
- Saglio G. N Engl J Med. 2010;362:2251-2259.