Protocol Number: 09-I-0184

Title:

Studies in the Pathogenesis of Systemic Capillary Leak Syndrome

Number:

09-I-0184

Summary:

Background:

- Systemic Capillary Leak Syndrome (SCLS) is a disorder of unknown cause characterized by episodes of life-threatening drop in blood pressure and leakage of fluids into tissues. The outcome from an episode of SCLS may be mild and resolve on its own, or may be severe and result in death. Although SCLS likely involves abnormalities in the cells lining blood vessels, the specific cause(s) of this disorder are not known.

- The treatment of choice for an acute SCLS episode is intravenous fluids and drugs such as norepinephrine (adrenaline), which are given to keep blood pressure at a level that will maintain vital organ function. This may be followed by a course of intravenous steroids. Currently, there is no cure or long-term effective preventive therapy for patients who experience recurrent episodes of SCLS.

- This protocol is focused on understanding what causes SCLS with the hope that research findings will lead to the design of safe and more effective treatments.

Objectives:

- To investigate mechanisms that may cause Systemic Capillary Leak Syndrome.

Eligibility:

- Patients between 16 and 65 years of age who have been diagnosed with SCLS.

- Patients must have a documented history of at least one episode of SCLS with all three of the following documented on at least one occasion: low blood volume, low blood pressure without cause, or evidence of protein leakage during the episode. Patients will also need to provide a letter of a referral from a treating physician.

Design:

- Patients will be evaluated at the National Institutes of Health (NIH) for approximately 4 to 5 days on an inpatient basis, and will undergo the following procedures:

--- Medical history and physical examination.

--- Blood samples for evaluation and research purposes, as well as possible genetic testing.

--- Apheresis procedure to obtain a larger volume of blood cells for research.

--- Bone marrow biopsy, if medically indicated.

--- Other medically indicated tests, such as skin tests to check for possible allergic reactions.

- Patients who have a capillary leak episode while at NIH will be treated with the standard of care for treating SCLS.

- Patients will be discharged from the protocol 2 weeks after the NIH visit.

- Relatives (who are between 1 and 80 years of age) of SCLS patients may also be asked to provide blood samples for the study.

Sponsoring Institute:

National Institute of Allergy and Infectious Diseases (NIAID)

Recruitment Detail

Type: Participants currently recruited/enrolled

Gender: Male & Female

Referral Letter Required: No

Population Exclusion(s): None

Eligibility Criteria:

INCLUSION CRITERIA FOR PARTICIPANTS:

Subject must be at least 16 years of age.

Having a diagnosis of SCLS after other diseases (e.g., sepsis, anaphylaxis, angioedema) were ruled out

Documented history of at least one episode of acute, reversible capillary leak as characterized by:

1. Hypotension (systolic blood pressure less than 90 mm Hg or diastolic blood pressure less than 60 mm Hg) or associated symptoms of hypotension (e.g., hypotonia [collapse], lightheadedness or syncope, incontinence, increased thirst)

2. Laboratory evidence of hemoconcentration (e.g., hemoglobin levels above the normal range [typically greater than17 g/dL], elevated leukocyte counts).

3. Clinical evidence of hypovolemia accompanying hypotensive symptoms such as fluid extravasation (e.g. edema) and/or laboratory evidence of protein extravasation such as serum hypoalbuminemia (less than 3.5 g/dL)

Letter of referral, with copies of pertinent medical history and laboratory studies, from prospective study subject's referring physician. Patients may self-refer, but a letter of referral from primary physician is still required.

Ability to give informed consent.

Willing to donate blood for sample storage to be used for future research.

INCLUSION CRITERIA FOR THE SUBJECT'S RELATIVES:

Ability to give informed consent.

Willing to donate blood for sample storage to be used for future research.

Subject's age can be between 2-80 years old.

EXCLUSION CRITERIA FOR PARTICIPANTS:

Presence of conditions which in the judgment of the investigator or the referring physician may put the subject at undue risk for travel (including frequent episodes of severe capillary leak symptoms not preventable by pre-medication, acute infection, severe thrombocytopenia [minimum platelet count of 30,000/microL], or significant cardiovascular disease)

Any condition that in the view of the principal investigator would make the subject unsuitable for enrollment in this study

EXCLUSION CRITERIA FOR SUBJECT'S RELATIVES:

Presence of conditions which in the judgment of the investigator or the referring physician may put the subject at undue risk for travel (including frequent episodes of severe capillary leak symptoms not preventable by pre-medication, acute infection, severe thrombocytopenia [minimum platelet count of 30,000/microL], or significant cardiovascular disease)

Any condition that in the view of the principal investigator would make the subject unsuitable for enrollment in this study

Special Instructions:

Currently Not Provided

Keywords:

SCLS

Recruitment Keyword(s):

Systemic Capillary Leak Syndrome

SCLS

Condition(s):

Systemic Capillary Leek Syndrome

Investigational Drug(s):

None

Investigational Device(s):

None

Intervention(s):

None

Supporting Site:

National Institute of Allergy and Infectious Diseases

Contact(s):

Patient Recruitment and Public Liaison Office
Building 61
10 Cloister Court
Bethesda, Maryland 20892-4754
Toll Free: 1-800-411-1222
TTY: 301-594-9774 (local),1-866-411-1010 (toll free)
Fax: 301-480-9793

Electronic Mail:prpl@mail.cc.nih.gov

Citation(s):

Cicardi M, Gardinali M, Bisiani G, Rosti A, Allavena P, Agostoni A. The systemic capillary leak syndrome: appearance of interleukin-2-receptor-positive cells during attacks. Ann Intern Med. 1990 Sep 15;113(6):475-7.

Atkinson JP, Waldmann TA, Stein SF, Gelfand JA, Macdonald WJ, Heck LW, Cohen EL, Kaplan AP, Frank MMSystemic capillary leak syndrome and monoclonal IgG gammopathy; studies in a sixth patient and a review of the literature. Medicine (Baltimore). 1977 May;56(3):225-39

Clarkson B, Thompson D, Horwith M, Luckey EH. Cyclical edema and shock due to increased capillary permeability. Am J Med. 1960 Aug;29:193-216.

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