Purified Type I Collagen Wound Matrix Improves Chronic Wound Healing in Patients with Recessive Dystrophic Epidermolysis Bullosa
2015; 32 (2): 220-225
Treatment of keratitis-ichthyosis-deafness (KID) syndrome in children: a case report and review of the literature
2015; 28 (2): 89-93
An open-label study to evaluate sildenafil for the treatment of lymphatic malformations
JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY
2014; 70 (6): 1050-1057
Recessive dystrophic epidermolysis bullosa is a severe genetic blistering skin condition resulting in chronic wounds. Nonhealing wounds were treated over 8 weeks using a reconstituted natural purified type I collagen skin substitute. Chronic wounds were defined as nonhealing wounds present for longer than 6 months. For each patient, two chronic wounds were identified and randomized into a control or treatment group. Both groups received standard-of-care wound dressings. The treatment group received an additional type I collagen skin substitute. Wound size was measured at baseline and weeks 1, 4, and 8. Pain, pruritus, and burning and stinging were assessed. Wound cultures were obtained at baseline and thereafter as was considered clinically relevant. Ten subjects were enrolled; seven completed the study. Six subjects showed a positive response to the type I collagen skin substitute. Three subjects demonstrated full wound reepithelialization. Wounds treated using the collagen skin substitute showed statistically significantly greater improvement. Average scores for pruritus and pain decreased significantly. Reconstituted natural purified type I collagen skin substitutes improved the healing of chronic wounds and may be a valuable addition to the epidermolysis bullosa wound care arsenal.
View details for DOI 10.1111/pde.12492
View details for Web of Science ID 000351747500017
View details for PubMedID 25557742
Rapidly Involuting Congenital Hemangioma Associated with Profound, Transient Thrombocytopenia
2014; 31 (3): 402-404
Lymphatic malformations can be challenging to treat. Mainstay interventions including surgery and sclerotherapy are invasive and can result in local recurrence and complications.We sought to assess the effect of 20 weeks of oral sildenafil on reducing lymphatic malformation volume and symptoms in children.Seven children (4 boys, 3 girls; ages 13-85 months) with lymphatic malformations were given oral sildenafil for 20 weeks in this open-label study. The volume of the lymphatic malformation was calculated blindly using magnetic resonance imaging performed before and after 20 weeks of sildenafil. Lymphatic malformations were assessed clinically on weeks 4, 12, 20, and 32. Both the physician and parents evaluated the lymphatic malformation in comparison with baseline.Four subjects had a lymphatic malformation volume decrease (1.0%-31.7%). In 2 subjects, despite a lymphatic malformation volume increase (1.1%-3.7%), clinical improvement was noted while on sildenafil. One subject had a 29.6% increase in lymphatic malformation volume and no therapeutic response. Lymphatic malformations of all 6 subjects who experienced a therapeutic response on sildenafil softened and became easily compressible. Adverse events were minimal.A randomized controlled trial will be necessary to verify the effects of sildenafil on lymphatic malformations.Sildenafil can reduce lymphatic malformation volume and symptoms in some children.
View details for DOI 10.1016/j.jaad.2014.02.005
View details for Web of Science ID 000336030400023
Multiple Eruptive Pilomatricomas in a 9-Year-Old Boy with Glioblastoma
2013; 30 (6): 756-758
Rapidly involuting congenital hemangioma (RICH) is an uncommon, often high-flow vascular tumor that presents at birth and involutes within the first year of life. It is clinically and histologically distinct from infantile hemangioma, kaposiform hemangioendothelioma, and tufted angioma, the latter two being associated with Kasabach-Merritt phenomenon. We present a female infant with RICH and profound, transient thrombocytopenia and review the extent and clinical course of thrombocytopenia in the context of congenital vascular tumors.
View details for DOI 10.1111/j.1525-1470.2012.01827.x
View details for Web of Science ID 000334884300044
View details for PubMedID 22937785
Clofarabine in refractory Langerhans cell histiocytosis
PEDIATRIC BLOOD & CANCER
2008; 51 (5): 703-706
A 9-year-old male presented to our dermatology clinic with a recent history of developing numerous cutaneous pilomatricomas, and was subsequently discovered to have sustained a recurrence of his glioblastoma multiforme. Immunohistochemical staining of a representative pilomatricoma and his original brain tumor revealed upregulation and nuclear localization of beta-catenin, a sign associated with poor prognosis in glioblastoma. We hypothesize that the development of multiple pilomatricomas may have been a hallmark of this patient's tumor recurrence and provide support for a recent report of an association between multiple pilomatricomas and gliomatosis cerebri.
View details for DOI 10.1111/j.1525-1470.2011.01714.x
View details for Web of Science ID 000326969100088
View details for PubMedID 22304393
Patients with multi-system Langerhans cell histiocytosis (LCH) who progress on frontline therapy have a dismal outcome. Responses to cladribine have been reported in relapsed LCH, but there are no well defined salvage regimens for LCH is refractory to therapy. The next generation deoxyadenosine analog, clofarabine, has demonstrated activity in patients with leukemia that is refractory to salvage regimens, including other nucleotide congeners; however, no experience exist on the use of clofarabine in LCH. In this report we describe significant single agent activity of clofarabine in disseminated LCH refractory to salvage regimens, including cladribine.
View details for DOI 10.1002/pbc.21668
View details for Web of Science ID 000259465400033
View details for PubMedID 18623218