Pegcetacoplan controls hemolysis in complement inhibitor-naive patients with paroxysmal nocturnal hemoglobinuria.

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease characterized by complement-mediated hemolysis. Pegcetacoplan is the first C3-targeted therapy approved for adults with PNH (United States), adults with PNH with inadequate response or intolerance to a C5 inhibitor (Australia), and adults with anemia despite C5-targeted therapy for ≥3 months (European Union). PRINCE was a phase 3, randomized, multicenter, open-label, controlled study to evaluate the efficacy and safety of pegcetacoplan vs control (supportive care only; eg, blood transfusions, corticosteroids, and supplements) in complement inhibitor-naive patients with PNH. Eligible adults receiving supportive care only for PNH were randomly assigned and stratified based on their number of transfusions (<4 or ≥4) 12 months before screening. Patients received pegcetacoplan 1080 mg subcutaneously twice weekly or continued supportive care (control) for 26 weeks. Coprimary end points were hemoglobin stabilization (avoidance of >1-g/dL decrease in hemoglobin levels without transfusions) from baseline through week 26 and lactate dehydrogenase (LDH) change at week 26. Overall, 53 patients received pegcetacoplan (n = 35) or control (n = 18). Pegcetacoplan was superior to control for hemoglobin stabilization (pegcetacoplan, 85.7%; control, 0; difference, 73.1%; 95% confidence interval [CI], 57.2-89.0; P < .0001) and change from baseline in LDH (least square mean change: pegcetacoplan, -1870.5 U/L; control, -400.1 U/L; difference, -1470.4 U/L; 95% CI, -2113.4 to -827.3; P < .0001). Pegcetacoplan was well tolerated. No pegcetacoplan-related adverse events were serious, and no new safety signals were observed. Pegcetacoplan rapidly and significantly stabilized hemoglobin and reduced LDH in complement inhibitor-naive patients and had a favorable safety profile. This trial was registered at www.clinicaltrials.gov as NCT04085601.

Incidence and radiographic findings of primary central nervous system lymphoma in immunocompetent patients in southern Thailand.

AIMS AND BACKGROUND: In the last two decades there have been conflicting reports concerning a rising incidence of primary central nervous system lymphoma (PCNSL) in immunocompetent patients. This study is being conducted to review the incidence and radiographic findings of PCNSL in a large tertiary care institution in southern Thailand. PATIENTS: A review was carried out in Songklanagarind University Hospital of the clinical records, CT and MRI images of immunocompetent patients who had histologically proven PCNSL diagnosed between January 1993 and December 2004. RESULTS: A total of 25 PCNSL patients were diagnosed over a 12-year period. The incidence trend was rising but not to a statistically significant extent. The median age at diagnosis was 57.4 years. Based on the Working Formulation classification, diffuse large cell was the most common subtype. All of the 20 patients with available immunohistochemical results had B-cell lymphomas. The radiographic findings in our series show that the majority of patients had a single lesion (60%) at a supratentorial location (50%). Most of the lesions were well circumscribed. Based on the density of the lesions before administration of a contrast medium, 56% of the lesions on CT images appeared hyperdense. On T1-weighted MRI images, the lesions of our patients were most frequently hypointense (67%) while the T2-weighted images were more commonly hyperintense (60%) and contrast enhancement was found in all lesions. There were 21 patients who received whole brain radiotherapy with doses ranging between 4.2 and 6.0 Gy. The median survival time was 14.4 months. CONCLUSIONS: This study indicates that there has been no significant increase in PCNSL cases over the last 12 years. The recognition of characteristic imaging features of PCNSL may facilitate a stereotactic procedure before steroid administration. In addition, we provide evidence that radiotherapy alone is insufficient in PCNSL.

Primary non-Hodgkin,s lymphoma of the uterine cervix.

Primary malignant lymphoma of the cervix is a rare disease. Because the number of reports of this cancer is limited, there is no consensus on its management, prognosis or the efficacy of various treatments. Primary malignant lymphoma of the cervix stage Ib was diagnosed in a 25-year-old woman. The patient was treated with 6 courses of CHOP chemotherapy (cyclophosphamide, doxorubicin, vincristine, and prednisone). Clinical and pathological responses were complete. This case supports current thinking in that, in selected young patients with primary malignant lymphoma of the cervix who desire to preserve fertility and ovarian functions, combination chemotherapy regimens such as CHOP are the treatment of choice.