3.3.2 Management Considerations
Without treatment, KLA commonly progresses and causes hemorrhage, effusions, cardiorespiratory compromise, multiorgan failure or even death. Unfortunately, there is no cure for KLA. Goal of therapy is to halt disease progression, control coagulopathy, manage symptoms and prevent complications. Currently, no universal guidelines exist for the treatment of KLA. KLA typically cannot be completely removed with surgery because of its infiltrative and multifocal nature and should be avoided given the high bleeding risk. Various medical therapies such as steroids, single or multiple chemotherapy agents, interferon, and sirolimus, have been used [9, 25-27]. Often used in combination as 2-drug or 3-drug regimens, oral sirolimus, intravenous or oral steroids, and vincristine, have become the preferred agents in the initial treatment of KLA [16]. Refer to Table 2 for suggested treatment regimen(s).
Bleeding complications are common in patients with KLA, but blood product replacement must be performed with caution. Platelet transfusions can worsen the underlying coagulopathy and oftentimes, does not improve platelet counts. Platelet transfusions should only be given for active, uncontrolled bleeding or immediately prior to or during a procedure with more than low risk for bleeding [27]. Fresh frozen plasma (FFP) or cryoprecipitate is recommended for hypofibrinogenemia less than 100 mg/dL. Prior to any procedure with more than minimal to low risk of bleeding and in patients with active bleeding, fibrinogen should be corrected to greater than 150 mg/dL. RBC transfusions do not have any negative effect on KLA, so should be given to maintain oxygenation, perfusion and symptomatic relief.
In our patient, platelet transfusions worsened her hypofibrinogenemia and failed to improve her thrombocytopenia. This is an expected phenomenon occurring because of increased platelet trapping in the KLA lesions and subsequent activation of the coagulation cascade. She was initially started on sirolimus but due to intolerable side effects was switched to the MEK inhibitor, trametinib. Her hematologic parameters normalized on sirolimus, and her disease remains controlled on trametinib.