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Oral smoothened inhibitor for advanced basal cell carcinoma of the hand: a case report.

Zhu GA, Chen A, Chang AL - Hand (N Y) (2014)

View Article: PubMed Central - PubMed

Affiliation: Department of Dermatology, Stanford University School of Medicine, 450 Broadway St., Redwood City, CA 94063 USA.

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Smoothened inhibitors have recently emerged as a nonsurgical, targeted therapy for treating advanced basal cell carcinomas (BCC)... This novel small-molecule drug class suppresses Hedgehog pathway signaling by binding the membrane protein, Smoothened... Over the course of treatment, the patient experienced commonly reported side effects, including hair loss and mild muscle cramping in the legs... He did not require treatment for his muscle cramping... Otherwise, the drug was well tolerated... In conclusion, we present this case to illustrate the utility of a new targeted therapy, oral Smoothened inhibitor treatment, for BCCs of the hand that are not amenable to surgical resection... Second, tumor shrinkage can be apparent early in the treatment course... Our patient's BCCs visibly decreased in size within 1 month of starting vismodegib but did not achieve maximal regression until 3 years of drug usage... Third, Smoothened inhibitor treatment can produce durable results without significant scarring or functional sequelae that might occur after surgical treatment... Further studies are under way to assess the use of Smoothened inhibitors to shrink BCCs preoperatively and/or in combination with other treatment modalities... In cases where Smoothened inhibitors alone fail to cure the disease or to arrest tumor growth, improved outcomes may be achieved with concomitant surgery or radiation therapy.

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a Bulky BCC tumors on dorsal hands and fingers prior to treatment with vismodegib. b After 4 weeks of vismodegib therapy, shrinkage of the BCCs was clinically evident. c After 6 months of vismodegib therapy, the BCCs continued to regress. d After 3 years of vismodegib therapy, the BCCs were nearly eradicated without surgery. a has previously been published in Gomez-Ospina et al [2]
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Fig1: a Bulky BCC tumors on dorsal hands and fingers prior to treatment with vismodegib. b After 4 weeks of vismodegib therapy, shrinkage of the BCCs was clinically evident. c After 6 months of vismodegib therapy, the BCCs continued to regress. d After 3 years of vismodegib therapy, the BCCs were nearly eradicated without surgery. a has previously been published in Gomez-Ospina et al [2]

Mentions: A 43-year-old Caucasian man with a known history of genetic predisposition to BCC presented with multiple large ulcerated, biopsy proven BCCs on his head, trunk, arms, and legs [2]. In particular, he noted the inability to type on a keyboard, drive a car, or carry out activities of daily living due to the large BCCs studding his dorsal hands, including some that traversed the joints. He had a 2.5-cm multilobular BCC on the distal tip of the left small finger (Fig. 1a).Fig. 1


Oral smoothened inhibitor for advanced basal cell carcinoma of the hand: a case report.

Zhu GA, Chen A, Chang AL - Hand (N Y) (2014)

a Bulky BCC tumors on dorsal hands and fingers prior to treatment with vismodegib. b After 4 weeks of vismodegib therapy, shrinkage of the BCCs was clinically evident. c After 6 months of vismodegib therapy, the BCCs continued to regress. d After 3 years of vismodegib therapy, the BCCs were nearly eradicated without surgery. a has previously been published in Gomez-Ospina et al [2]
© Copyright Policy - OpenAccess
Related In: Results  -  Collection

Show All Figures
getmorefigures.php?uid=PMC3928376&req=5

Fig1: a Bulky BCC tumors on dorsal hands and fingers prior to treatment with vismodegib. b After 4 weeks of vismodegib therapy, shrinkage of the BCCs was clinically evident. c After 6 months of vismodegib therapy, the BCCs continued to regress. d After 3 years of vismodegib therapy, the BCCs were nearly eradicated without surgery. a has previously been published in Gomez-Ospina et al [2]
Mentions: A 43-year-old Caucasian man with a known history of genetic predisposition to BCC presented with multiple large ulcerated, biopsy proven BCCs on his head, trunk, arms, and legs [2]. In particular, he noted the inability to type on a keyboard, drive a car, or carry out activities of daily living due to the large BCCs studding his dorsal hands, including some that traversed the joints. He had a 2.5-cm multilobular BCC on the distal tip of the left small finger (Fig. 1a).Fig. 1

View Article: PubMed Central - PubMed

Affiliation: Department of Dermatology, Stanford University School of Medicine, 450 Broadway St., Redwood City, CA 94063 USA.

AUTOMATICALLY GENERATED EXCERPT
Please rate it.

Smoothened inhibitors have recently emerged as a nonsurgical, targeted therapy for treating advanced basal cell carcinomas (BCC)... This novel small-molecule drug class suppresses Hedgehog pathway signaling by binding the membrane protein, Smoothened... Over the course of treatment, the patient experienced commonly reported side effects, including hair loss and mild muscle cramping in the legs... He did not require treatment for his muscle cramping... Otherwise, the drug was well tolerated... In conclusion, we present this case to illustrate the utility of a new targeted therapy, oral Smoothened inhibitor treatment, for BCCs of the hand that are not amenable to surgical resection... Second, tumor shrinkage can be apparent early in the treatment course... Our patient's BCCs visibly decreased in size within 1 month of starting vismodegib but did not achieve maximal regression until 3 years of drug usage... Third, Smoothened inhibitor treatment can produce durable results without significant scarring or functional sequelae that might occur after surgical treatment... Further studies are under way to assess the use of Smoothened inhibitors to shrink BCCs preoperatively and/or in combination with other treatment modalities... In cases where Smoothened inhibitors alone fail to cure the disease or to arrest tumor growth, improved outcomes may be achieved with concomitant surgery or radiation therapy.

No MeSH data available.


Related in: MedlinePlus