Lamellar ichthyosis (LI) is a genetically inherited disorder of keratinization. LI is a very disabling stigmatizing skin disorder due to the presence of generalized dark scales with a dirty appearance and the chronic course of the disease. Current therapy consists of topical emollients and keratolytics and systemic retinoids, but always with a palliative approach. Therefore, new safe and efficacious therapeutic alternatives are always welcome.

Isolated papers have recently communicated the efficacy of topical 10% N-acetylcysteine (NAC) in the management of LI. NAC is an aminoacid derivative with an antiproliferative effect on fibroblasts and keratinocytes. Interestingly, an important reduction in scaling and improvement of symptoms was documented after only several weeks of therapy. NAC is labile and releases sulphur-containing compounds. Therefore, malodour is the main handicap of the product. Several recommendations have been reported to reduce this disadvantage: formulating NAC in a water-in-silicone emulsion which makes the formula more stable and adding aromatic substances such as orange essence. Moreover, some patients experience local irritation, burning and pruritus, minor adverse events which can be improved by tapering the NAC concentration to 5%, or applying the drug less frequently. No systemic side effects have been reported.

Of course, although more studies are needed in order to determine the role NAC may play in the treatment of hyperproliferative skin disorders, this seems to be a very attractive new therapeutic option in the difficult field of ichthyosis.

Do you have any personal experience with this new treatment?.

Successful treatment with topical N-acetylcysteine in urea in five children with congenital lamellar ichthyosis.

Systemic propranolol is currently a widespread treatment when managing infantile hemangiomas (IH), but what about topical propranolol?. Little has been reported regarding this indication. In a very interesting paper just published by Dr. Guangqi Xu et al in the Journal of the American Academy of Dermatology the authors evaluate the efficacy and safety of topical propranolol in the treatment of superficial IHs.

The authors communicate their retrospective experience in 25 patients suffering from a total of 28 superficial IHs. 1% propranolol ointment was applied three times a day, rubbed on the lesion, during a mean duration of 21 weeks. The median age of the patients was 4 months. A good response to therapy was documented in 16 IHs (57%), a partial response in 9 IHs (33%) and no response in 3 IHs (10%). Interestingly, therapy seemed to be more efficacious in younger patients. Based on this last finding the authors suggest that treatment was more effective in the proliferation phase than in the maturation phase. No significant side effects were detected.

Of course, the authors underline that this is a small retrospective study and therefore, prospective, controlled, randomized studies are needed to define the exact role topical propranolol should play in the management of superficial IHs.

Dr. Xu an co-workers confirm previous reports: according to their results 1% propranolol ointment is effective and safe in superficial IHs and the authors support its use as adjuvant treatment during the usual wait-and-see period.

Topical propranolol for treatment of superficial infantile hemangiomas.

Xu G, Lv R, Zhao Z, Huo R.

J Am Acad Dermatol. 2012 Apr 17.  

PMID:

22516113

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

More studies are needed…

Dr. Cynthia J. Price and co-workers have just published a very interesting study in the Archives of Dermatology focusing on the controversy propranolol vs corticosteroids when treating infantile hemangiomas (IHs):

Propranolol vs Corticosteroids for Infantile Hemangiomas: A Multicenter Retrospective Analysis.

Price CJ, Lattouf C, Baum B, McLeod M, Schachner LA, Duarte AM, Connelly EA.

Arch Dermatol. 2011 Dec;147(12):1371-6. PMID: 21844428

IHs are frequent, and they can pose challenging clinical scenarios due to bleeding, ulceration and compression of vital structures. In addition, they can have an important cosmetic impact and worsen life quality. When these reasons motivate treatment, corticosteroids used to be the mainstay of therapy, but since 2008 more and more clinicians are in favour of using propranolol as an alternative.

The authors performed a multicenter retrospective study including 110 patients with IHs, with the objective to determine whether propranolol is safe, effective and superior to oral corticosteroids when managing IHs. 68 patients received propranolol with a mean duration of treatment of 7.9 months, and 42 patients received oral corticosteroids with a mean duration of treatment of 5.2 months. The main results the authors underline are the following:

1. Efficacy: 82% of the patients in the propranolol group achieved clearance of 75% or more, while only 29% of the patients in the corticosteroid group obtained that result (P<.01).
2. Security: only 1 patient under propranolol developed an adverse effect (hypoglycemia), while every patient in the corticosteroid group (42/42) suffered from 1 or more adverse effects (P<.01).
3. Surgery due to insufficient response or unacceptable cosmetic outcome: propranolol reduces surgical referrals as only 12% of the patients in the propranolol group needed surgery after treatment compared with 29% of the patients in the corticosteroid group (P=.03).
4. Ulceration: 26% of the IHs under oral corticosteroids ulcerated, but ulceration was only observed in 6% of the cases under propranolol (P<.01).

Based on their findings Dr. Cynthia J. Price et al recommend propranolol as a first-line agent when treating IHs due to its safety and efficacy profile. Although retrospective, this is a very interesting article due to the lack of large published studies regarding this issue. 

Intensive topical treatments are probably underused as first-line approach for severe atopic dermatitis flares. In the last October issue of the Journal of the American Academy of Dermatology, Dr. Tushar S. Dabade and co-workers have published an original paper reviewing the efficacy of intensive topical treatments for pediatric atopic dermatitis.

Wet dressing therapy in conjunction with topical corticosteroids is effective for rapid control of severe pediatric atopic dermatitis: Experience with 218 patients over 30 years at Mayo Clinic.

Vitiligo is a very common skin disorder in infancy, but, is childhood vitiligo similar to vitiligo affecting adult patients?.  Few papers have compared vitiligo presenting before the age of 12 years, or childhood-onset vitiligo (COV), with later-onset vitiligo (LOV). In a recent issue of the Journal of the American Academy of Derrmatology, Dr. Electra Nicolaidou and co-workers have published a very interesting study analyzing the epidemiologic and clinical characteristics of COV, when compared with LOV:

Childhood- and later-onset vitiligo have diverse epidemiologic and clinical characteristics.

The authors performed a cross-sectional study enrolling two groups of consecutive patients from January 2005 to December 2009. 126 patients were included in the COV group, and 107 in the LOV group.

According to their results,  Dr. E. Nicolaidou and co-workers conclude that the main learning points of the study are:

1. Disease presentation: there were statistically significant differences between both groups regarding sites at initial presentation. COV had a predilection for the eyelids and lower extremities, while LOV frequently presented involving the upper extremities, and particularly the hands. More cases of segmental vitiligo at initial presentation were documented in the COV group. In addition, COV was less related to stressful events at presentation.
2. Disease progression: a progressive course of the disorder was documented in the majority of patients from both groups, but COV progressed at a slower rate. Vitiligo vulgaris was the most common disease type both groups progressed to. 
3. Associated endocrine diseases: COV presented with a lower prevalence of thyroid diseases than LOV. When performing a multivariate analysis, the variables that were associated with thyroid disease in the context of COV were family history of thyroid disease and longer duration of vitiligo.
4. Family history: a family history of vitiligo was reported in both groups (35% of COV and 33% of LOV).
5. Study limitations: possible selection bias enrolling more severe cases and possible recall errors.

Dr. Nicolaidou et al end their article reminding us that children with vitiligo are at risk of developing thyroid disease in their lives, mainly if there are other cases of thyroid disease within the family.