Thrombotic thrombocytopenic purpura - analysis of clinical features, laboratory characteristics and therapeutic outcome of 24 patients treated at a tertiary care center in Saudi Arabia

Objective: Thrombotic thrombocytopenic purpura [TTP] is a life-threatening disease. The primary aim was overall response rate [ORR] assessment in the treated patients

Methods: This retrospective study included 24 patients treated during 2006-2015. TTP patients with microangiopathic hemolysis [MAHA] and thrombocytopenia were included. We analyzed clinical features, laboratory characteristics and treatment outcomes of 24 TTP patients treated at our tertiary care center [KFMC]

Results: Twenty-four TTP patients [18 females; 6 males] had a mean age of 33.5 +/- 13.9 years; 22[91%] had neurologic features, 7[29%] fever, 10[42%] renal impairment; 4[20.83%] cardiac manifestations; 22[91.7%] had triad with additional neurologic abnormalities; only 2[8.2%] had pentad of TTP. Majority [54.16%] had idiopathic TTP. All patients received therapeutic plasma exchange [TPE]; 23[95.8%] received adjunctive corticosteroids and 13[54.2%] received rituximab either due to refractoriness to TPE on tilde day7, or earlier. Twenty-one out of 24 [87.5%] achieved complete remission [CR] without any subsequent relapse. At 22 months [median, range 1-113], 20 patients [83.3%] are alive at the time of report. Three patients died during acute episode because of sever disease or delayed treatment and one died in CR

Conclusion: TPE, steroids and or rituximab was very effective in preventing high risk of mortality and achieving durable CR in 87.5% of patients. More awareness is needed for early diagnosis and early referral to centers with appropriate tertiary care facilities

Cold agglutinin disease in fibrolamellar hepatocellular carcinoma: a rare association with a rare cancer variant

Cold agglutinin disease [CAD] is a rare autoimmune hemolytic anemia. Although it can occur secondary to lymphoproliferative disorders and autoimmune or infectious diseases, CAD is rarely reported as secondary to solid tumors. We report a case of a woman aged 18 years diagnosed with a well-differentiated hepatocellular carcinoma of the fibrolamellar subtype, who was shown to have CAD also. Her general condition, including CAD, improved after targeted therapy with sorafenib for the hepatocellular carcinoma and only conservative measures for the CAD that consisted of avoidance of cold. In summary, although it is an extremely rare association and less common than lymphoproliferative disorders, CAD can be associated with solid tumors