Left, a large ulceration involving the right medial foot and ankle with noninflammatory rolled borders. This ulcer was treated empirically for years as a venous stasis ulcer until biopsy revealed it was, in fact, basal cell carcinoma.
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Right, sagittal T1-weighted magnetic resonance imaging revealed invasion of mass into the anterior joint space and soft tissues around the flexor digitorum tendon and neurovascular bundles arrows. In most cases, surgical excision is curative, but a subset of patients have inoperable, locally advanced, or metastatic disease that drastically limits treatment options.
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The median survival in metastatic basal cell carcinoma is 24 months, and conventional chemotherapy has not been shown to improve the prognosis. In addition to the burden of sporadic basal cell carcinoma, patients with the rare autosomal-dominant genetic disorder basal cell nevus syndrome Gorlin syndrome develop multiple basal cell lesions over their lifetime. The syndrome may also involve abnormalities of the skeletal system, genitourinary tract, and central nervous system, including development of medulloblastoma.
The lesions occur in multiple numbers and can develop anywhere on the body, including on non—sun-exposed areas. Patients who have Gorlin syndrome need meticulous monitoring every 2 to 3 months so that basal cell lesions can be recognized early and treated before they become locally advanced.
Keeping up with the numerous medical appointments and invasive treatments can be physically and mentally taxing for patients. In , Robarge et al 6 described a potent inhibitor of the hedgehog pathway that was later optimized for potency and desirable pharmacologic traits, resulting in the drug vismodegib. Two phase 2 multicenter clinical trials 9,10 of vismodegib were published in In the first, which was not randomized, 9 33 patients with metastatic basal cell carcinoma and 63 patients with locally advanced disease were treated with vismodegib.
In the second trial, 10 patients with Gorlin syndrome were randomized to either vismodegib 26 patients or placebo 16 patients. No tumors progressed in the treatment group. Results in some patients were dramatic, with complete healing of large ulcerative tumors. The trial was ended early in view of significant efficacy in the treatment group. Unfortunately, vismodegib has significant drawbacks.
Although these effects were considered mild or moderate, they tended to persist, and almost every patient developed at least one. One possibility is to use the drug for a few months to shrink tumors to the point that they become eligible for surgery.
This is especially important for high-risk tumors, such as those near the eye or other vital structures. Another option is to combine vismodegib with other agents—either new ones on the horizon or existing nonspecific medications. For example, the antifungal itraconazole has been shown to inhibit hedgehog signaling and perhaps could be combined with vismodegib to increase response and reduce resistance.
Finally, a topical or intralesional form of vismodegib would be useful not only to reduce systemic toxicity, but also to increase efficacy when combined with other topical or systemic medications. Case 2. A year-old woman presents with worsening psoriasis. What are her options? Figure 2. Sometimes, a biologic is prescribed to treat a child who has psoriasis. This can be very effective for a child who has severe psoriasis. The FDA has approved usetekinumab to treat people 12 years of age and older who have moderate-to-severe psoriasis.
For this reason, dermatologists carefully screen each patient before prescribing a biologic. Blood tests and tuberculosis TB testing are typically required. Some patients need additional medical tests. For many people with moderate-to-severe psoriasis or psoriatic arthritis, a biologic may offer the most effective treatment available. If you take a biologic continuously, it tends to be more effective.
Stopping and starting can cause a biologic to lose its effectiveness and may cause certain side effects. If this happens, another biologic may work. While a biologic can lose its effectiveness over time, studies show that for many people a biologic remains an effective and safe treatment for years.
It is important to know that no one biologic works for everyone. One biologic could fail to help you, but another could work very well. This varies with the type of biologic. Some shots you can give yourself at home, after learning how to give yourself the shot.
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How often you take the biologic varies from twice a week to once every three months. Your dermatologist will tell you how often you should take it. Be sure your dermatologist knows all of the medicines you take, including ones that you can buy without a prescription. Each biologic has its own list of possible side effects. Most are mild and do not cause patients to stop taking the biologic.
Some of the more common side effects include:. Because the biologics work by calming down part of your immune system, anyone taking a biologic has an increased risk of developing a serious infection. The risk is higher in patients who have diabetes, smoke or chew tobacco, or have a history of infections.
Older patients also have a higher risk. Dermatologists watch their patients for signs of problems. For both formats the functionality available will depend on how you access the ebook via Bookshelf Online in your browser or via the Bookshelf app on your PC or mobile device.
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Comprehensive Dermatologic Drug Therapy
Close Preview. Toggle navigation Additional Book Information. Summary The Handbook of Systemic Drug Treatment in Dermatology helps prescribers and patients make rational decisions about drug treatment while considering known risks and potential unwanted effects. Editor s Bio Edited by Sarah H. Reviews "This nearly pocket-sized book is an excellent summary and simplification of the use of systemic medications commonly practiced by dermatologists.
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