More case studies:
I'd now like to introduce Dr. Madeleine Duvic from the M.D. Anderson Cancer Center who will discuss her experiences with Targretin.
DR. SCHILSKY: I'd just like to remind the sponsor that you've already exceeded your allotted time. So, I'd ask that you either abbreviate this presentation or move directly to your conclusions. Thank you.
DR. DUVIC: Thank you. My name is Madeleine Duvic, and I've been involved with the care of CTCL patients at M.D. Anderson since 1985. We actively treat over 600 patients and evaluate 100 new patients per year, and 41 percent of my patients have been treated in the Targretin trial.
I would like to share with you the dramatic and long-lasting improvement seen in four elderly patients.
First was a 71-year-old man with stage IIA mycosis fungoides for 13 years who had failed 9 previous therapies, including several combinations of chemotherapy and pentostatin. He had 59 percent patch and plaque involvement. The patches are not shown well in the global photograph. But at 9 months, you can see he has a complete remission.
However, there's an index lesion here that did not resolve and is shown in the next slide and remains to this day.
Again, the CA in yellow and the PGA in green are back to back and show a response of 50 percent as early as week 4.
This patient had a 98 percent response, disappearance of all cutaneous lesions, resolution of adenopathy and normalization of Sezary cell counts to 0 by week 12. He has had only triglyceridemia as a side effect, and he's been in almost complete remission for over 17 months.
Secondly, an 81-year-old man with 7 years of CTCL and 7 previous therapies presented with 48 percent thick plaques on the body and adenopathy. He resolved with only residual hyperpigmentation on the trunk and on the extremities.
His skin biopsy at baseline has shown resolution of the dermal infiltrate by week 8 and normalization of the epidermal changes.
Again, the CA and PGA were similar confirming PR at 4 weeks and CR at 12 weeks.
To summarize, this patient had a complete response confirmed by biopsy, resolution of nodes and Sezary cells which is ongoing at 17 months. Only mild side effects were present.
The third case is a 63-year-old male with a 5-year history of CTCL who submitted a letter. He developed a large tumor with large cell transformation that relapsed on both CMED and ESHAP chemotherapies. This tumor resolved by week 4, leaving only an ulceration, and healing with normal skin that remains to this day.
He also had clearance of 39 percent of his body involved with patch/plaque disease. Again, a biopsy comparing week 8 to week 0 shows resolution of the dermal infiltrate, resolution of the ulceration, and normalization.
Again, his response was rapid, as shown by both the CA and the PGA, and he continues in almost complete remission ongoing at 2 years.
Finally, we saw patients with exfoliative erythroderma such as this 71-year-old man with Sezary syndrome who had at baseline 100 percent body surface area and lichenified skin. He had failed interferon, photopheresis, nitrogen mustard.
This patient was actually classified as a progressive disease in the study because lymphadenopathy noted prestudy was not appreciated at baseline and reappeared at week 4 evaluation. Since he had no index lesions, his assessment can only be determined by PGA, and it reached 50 percent by week 24 with sustained improvement shown at week 40 on the next slide.
This man has skin like an alligator. It was thick, scaly, with lichenification of all his extremities, and over the course of therapy, his skin completely normalized, returning to normal color which remained after study.
In summary, excellent clinical responses are seen for oral Targretin at all stages of this disease. As shown, the composite assessment was overly conservative and underestimated the clinical responses seen in some patients.
Targretin has important advantages over other available agents. As an oral capsule, it does not require venous access or catheters.
Patients do not get infections resulting from lines or from treatment.
Targretin is not immunosuppressive.
The responses to Targretin are rapid, dose-related, and durable. Side effects are reversible and can be prevented, treated, and monitored. From my experience, Targretin is an important new therapy for CTCL. Targretin would be helpful and a welcome addition for treating CTCL at all stages of the disease. Many of the patients I treat have run out of available or non-immunosuppressive options.
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