I’m extremely pleased to report that Dr. Kirk Druey and his team from the U.S. National Institutes of Health (NIH) have had their paper based on the results of a patient survey on the outcome of IVIG as therapy for SCLS accepted for publication later this year in _The American Journal of Medicine_.
The peer-reviewed and accepted, but not yet edited, typeset or proofed, version is available online for journal subscribers, or others with access (e.g., through a university or public library subscription), to the Journal's restricted website. Those Community members who lack access and wish to receive this pre-publication version of the article may request it from me at _aporzeca@american.edu_ Please include your RareShare username in the email to me.
As many of you will recall from a post earlier this year (see Discussion Forum topic "Survey of All Users of IVIG"), the article reflects the results of a questionnaire sent by Dr. Druey to all SCLS patients who have been in touch with him over the years, to find out about their experience with any IVIG therapy. Most of the questionnaire respondents, of course, are members of this Community.
To quote from the article's Conclusion:
"Our case series, a European registry, and numerous individual case reports suggest that monthly administration of IVIG is associated with a marked reduction in the number of episodes of hypovolemic shock and edema in patients with classic acute Systemic Capillary Leak Syndrome. These studies indicate that IVIG may be considered as frontline therapy for subjects with a clear-cut diagnosis of the Systemic Capillary Leak Syndrome and recurrent attacks."
*Therefore, this article is likely to promote the use of, and facilitate access to, IVIG as the medication of choice for all confirmed SCLS patients.*
Following are the results as summarized in the abstract appearing on the Internet page
_http://www.ncbi.nlm.nih.gov/pubmed/25193271_
*High dose intravenous immunoglobulin therapy of the Systemic Capillary Leak Syndrome (Clarkson disease).*
Xie Z, Chan E, Long LM, Nelson C, Druey KM.
Laboratory of Allergic Diseases, NIAID/NIH, Bethesda, MD 20892
BACKGROUND:
The Systemic Capillary Leak Syndrome is a highly rare disorder of unknown etiology. The disease is characterized by episodes of transient vascular collapse, which leads to hypotensive shock and anasarca. Previous treatment of this potentially devastating condition has been largely ineffective. We evaluated IVIG prophylactic therapy in a cohort of 29 patients with Systemic Capillary Leak Syndrome in a longitudinal follow-up study.
METHODS:
All patients received treatments at the discretion of their primary providers and retrospectively via questionnaire-recorded symptoms beginning with their first documented episode of the Systemic Capillary Leak Syndrome until May 31, 2014.
RESULTS:
Twenty-two out of 29 patients responded to the questionnaire, and 18 out of the 22 respondents received monthly prophylaxis with IVIG during the study period for a median interval of 32 months. The median annual attack frequency was 2.6/patient prior to IVIG therapy and 0/patient following initiation of IVIG prophylaxis (P = 0.001). 15 out of 18 subjects with a history of one or more acute Systemic Capillary Leak Syndrome episodes experienced no further symptoms while on IVIG therapy.
CONCLUSIONS:
*IVIG prophylaxis is associated with a dramatic reduction in the occurrence of Systemic Capillary Leak Syndrome attacks in most patients, with minimal side effects.* A prospective, randomized trial may be necessary to fully assess the benefits of IVIG for the Systemic Capillary Leak Syndrome and to determine optimal dosage and duration of therapy.