A novel combined treatment for plasminogen deficiency with lung involvement

Abstract

Plasminogen deficiency is characterized by fibrin‐rich pseudomembrane formation on all mucosal surfaces, particularly the conjunctiva. Respiratory system involvement is common; fibrin often obstructs the upper or lower respiratory tract, causing death. Although many treatments have been applied, no definitive treatment (especially of the respiratory involvement) yet exists. Although excision of tracheobronchial tree membranes affords temporary improvement, this should be performed only for patients in severe respiratory distress; the procedure triggers fibrin redeposition.Here, we share our experience with an 8‐year‐old plasminogen‐deficient female with severe lung involvement; we successfully delivered local tissue plasminogen activator followed by nebulized fresh frozen plasma.

A Successful Treatment Approach in 2 Patients With Type 1 Plasminogen Deficiency: Intratracheal Application of Fresh Frozen Plasma and Tissue Plasminogen Activator

“…Patients face an increased risk of morbidity and mortality and their quality of life is significantly affected 1 . RSI occurs in 20% of patients and is the most important cause of mortality 4,8,13 . Fibrin plaques triggered by infection or local tissue damage accumulate in the mucosa of the respiratory system and cause upper and lower respiratory tract symptoms.…”

“…Recently, intratracheal tPA and FFP have been used in the treatment of patients with severe respiratory distress and have been successful 4,8,12 . The main goal here is to replace the PD with FFP and to dissolve fibrin plaques by converting plasminogen to plasmin and causing fibrinolysis with tPA.…”

“…For this purpose, patients were started to receive FFP intravenously at 10 to 15 mL/kg/d to increase blood plasminogen levels. On the sixth day of FFP administration, the protocol applied by Kilinc et al 8 to a patient with PD lung involvement was applied with bronchoscopy. Membrane excision with rigid bronchoscopy was performed.…”

“…Even though excision of the membranes in the tracheobronchial tree provides a temporary improvement, fibrin formation during the healing process results in the recurrence of respiratory symptoms. In recent years, there have been cases with successful outcomes with systemic and bronchoscopically administered fresh frozen plasma (FFP) and tissue plasminogen activator (tPA) 4,7,8 …”

A Peculiar Disease of a Young Woman who Wants to Get Pregnant

“…As plasma-derived plasminogen concentrate (NCT02312180) was not available in Turkey, fresh frozen plasma (FFP) was given both intravenously and directly into the uterine cavity in an off-label way. Our clinical practice with FFP and alteplase, a recombinant human tissue-type plasminogen activator (rTPA) administered by bronchoscopic method, was published recently [ 7 ]. Therefore, a similar regimen was utilized for this case.…”