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Lymphangioleiomyomatosis (LAM) Related to Pulmonary Hypertension

N. Gourineni

A. Janakiraman

S. Soma

George Sokos

Srinivas Murali


Raymond Benza


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Conference: 2010 International PHA Conference and Scientific Sessions

Release Date: 06.24.2010

Presentation Type: Abstracts

Gourineni N, Janakiraman A, Soma S, Sokos G, Murali S, Benza R.
The Gerald McGinnis Cardiovascular Institute at Allegheny General Hospital, Pittsburgh, PA, USA

BACKGROUNDTuberous Sclerosis (TSC) manifests as a neurocutaneous disorder. Lung involvement is considered rare. Lymphangioleiomyomatosis (LAM), most commonly seen in women of childbearing age, is the major pulmonary disorder seen in TSC. Unlike TSC, LAM is thought to be hormone-dependent, specifically estrogen mediated, which explains the rarity of its occurrence among post-menopausal women and men. At times, Pulmonary Hypertension (PH) can be seen in this scenario, although little is known about its response to conventional therapy.

CASE SUMMARY: A 61 year old nulliparous post-menopausal woman with history of TSC presented to our hospital with progressive dyspnea. She was noted to be in New York Heart Association (NYHA) class IV heart failure and a transthoracic echocardiogram showed estimated pulmonary artery pressure (PAP) of 100mmHg. A right heart catheterization (RHC) confirmed PH with PAP of 141/64 with a mean of 94. After reviewing the literature we treated her PH with IV treprostinil and oral sildenafil. Her right ventricular failure was stabilized with inotropes and intravenous (IV) diuretics. Additional LAM therapy was initiated which consisted of rapamycin, doxycycline, erythromycin and statin therapy; all of which focus on various signaling pathways of estrogen antagonism of LAM cells. Within 8 weeks, there was remarkable improvement in her exercise capacity (six minute walk distance - 6MWD) from baseline of 56 meters to 256 meters in addition to her improvement of NYHA functional status from class IV to class II. In four months, she had sustained treatment efficacy with hemodynamics improving from a pulmonary vascular resistance (PVR) of 1783 dyn.sec/cm5 to 400 dyn.sec/cm5 and exercise capacity further improving to 352 meters.

 

Time (Months) 0 (November) 4 months (March)
NYHA Functional Class IV II
6MWD (meters) 56.12 360
Treprostinil IV (ng/mg/kg) 32 42
Sildenafil 60 mg/day (divided doses) 60 mg/day (divided doses)
Rapamycin 3 mg/day (divided doses) 1 mg/day

Hemodynamics:
     Right Atrial Pressure (cm H2O)
     Mean PAP (mm Hg)
     Fick Cardiac Index
     PVR (Wood units)


18
99
1.6
22.2


7
40
3.9
5.0

 

CONCLUSIONS: Standard evidence based therapy for pulmonary arterial hypertension can be applied to PH related to LAM with good results. Sequential combination therapy to treat LAM specifically can also be applied and may offer additional benefit.