Peer-review started: November 5, 2017
First decision: November 20, 2017
Revised: November 27, 2017
Accepted: December 6, 2017
Article in press: December 6, 2017
Published online: February 2, 2018
Processing time: 124 Days and 4.2 Hours
To systematically review literature for management of alpha-1 antitrypsin deficiency (AATD) panniculitis.
Multiple databases were searched using combinations of pertinent terms. Articles were selected describing panniculitis treatment in patients with AAT < 11 μmol and/or PiZZ genotype, with no language limitation. All relevant articles were accessed in full text. Independent review of abstracts and full manuscripts was conducted by 2 reviewers, and quality assessment by one reviewer (checked by a second). Data extraction was conducted by one reviewer (checked by a second). Narrative synthesis only was conducted, as data were unsuitable for meta-analysis.
Thirty-two case reports and 4 case series were found. Augmentation therapy (infusions of plasma-derived AAT) was the most successful, with complete resolution of symptoms in all patients. Dapsone is a less expensive option, and it achieved clinical resolution in 62% of patients, but it is very poorly tolerated. Among other single-agent antibiotics, doxycycline was the most successful with complete clinical resolution seen in 33% of patients. Immunosuppressants were largely unsuccessful; 80% of patients exhibited no response. Liver transplantation and therapeutic plasma exchange displayed complete resolution in 66% of patients. Other strategies, such as non-steroidal anti-inflammatory drugs or antibiotics other than dapsone did not show sufficient response rates to recommend their use. Authors note the risk of bias imposed by the type of evidence (case reports, case series) available in this field.
Dapsone is the recommended first line therapy for AATD panniculitis, followed by augmentation therapy. Plasma exchange may be an alternative in the setting of rapidly progressive disease.
Core tip: There is limited evidence regarding treatment of alpha-1 antitrypsin deficiency (AATD)-related panniculitis. This study aims to systematically review literature for management of AATD-related panniculitis. The most effective treatment is augmentation therapy. Dapsone shows moderate success, but is significantly cheaper and easier to administer. Other treatments have shown limited benefit. Recommended first line therapy for AATD-related panniculitis is dapsone, followed by augmentation therapy. Plasma exchange may be an alternative in the setting of rapidly progressive disease, if augmentation were not available.