Off label, on point
Spironolactone can be an effective treatment for patients with acne.

Spironolactone is primarily used for the treatment of heart failure, high blood pressure, or hypokalemia — but more than a few dermatologists know that it can also be a secret weapon for treating acne in women.
Emmy Graber, MBA, MD, FAAD, founder and director of the Dermatology Institute of Boston, said that in spite of it being used for acne treatment for years, some dermatologists remain a little uncertain about using it today.
“It has FDA approval for other indications, but not for treating acne, and for that reason, some dermatologists are a little hesitant to use it for acne because it is an off-label use,” she said.
The use of spironolactone was explored by Dr. Graber and other presenters, including Hilary Baldwin, MD, FAAD, Julie Claire Harper, MD, FAAD, and Bethanee Jean Schlosser, MD, PhD, FAAD, in Friday's session, F021 – Practical Guidelines for Using Spironolactone in Acne Patients.
Pinpointing the patient
One of the keys to effectively using the drug, Dr. Graber explained, is knowing who will benefit the most. For example, she said spironolactone is primarily used in women because of its potential side effects (such as gynecomastia) in men. But there are other factors to consider in deciding who is — and isn’t — a good candidate.
“You have to look at the age of the patient and when we might start treatment,” Dr. Graber said. “It also depends on what type of acne they have. We used to think that patients who were good candidates had primarily lower face or jawline acne, but that’s not necessarily the case anymore.”
Dr. Graber said dermatologists also must know when it is appropriate to do a lab workup in patients who may benefit from spironolactone treatment.
“Do we think that everyone whose acne flares up around the time of the menstrual cycle needs a hormonal (lab) workup for that acne?” she asked. “Probably not, but there are some telltale signs to look for, such as hirsutism, a deepening of the voice, and irregular periods, which may warrant a laboratory workup.”
Consider the side effects
As with any drug, there are potential side effects to consider with spironolactone, such as irregular menstrual cycles and the potential for hyperkalemia.
Dr. Graber said there has long been concern over the possibility of breast cancer as well.
“Some physicians are leery of putting patients on spironolactone because of breast cancer concerns, but this is not supported by scientific studies,” she said.
Ultimately, Dr. Graber said it is up to dermatologists to decide if spironolactone is right for them and for their patients. But whether they use it or not, they should at least be aware of its potential and its benefits to the medical community.
“It is of particular interest to dermatologists because, although it is off-label, it can be a very successful treatment for acne,” she said. “In this era of antibiotic stewardship, we’re using antibiotics more selectively these days, so we want alternatives, and this is a non-antibiotic alternative that dermatologists should consider. It’s been proven to be an effective drug for treating acne.”