Bilateral subdural hematomas, an unusual vaso-occlusive event and prolonged prothrombin time in a 58-year-old victim of an armed robbery.

Factor VII deficiency is one of the most common of rare bleeding disorders(1). This autosomal recessive disorder has a prevalence of 1:500,000 with geographic variations. Clinical manifestations vary from asymptomatic to severe mucocutaneous bleeding. According to the International Registry of Factor VII Deficiency (IRF7) epistaxis is the most common clinical manifestation. Gastrointestinal and central nervous system(CNS) bleeding are rare presentations.(2-4) We present here the case of a patient with life-threatening CNS bleeding who was found at the age of 58 years to have congenital factor VII deficiency.

Phase II trial of gemcitabine as first line chemotherapy in patients with metastatic or unresectable soft tissue sarcoma.

OBJECTIVE: The availability of effective chemotherapy agents or regimens for soft tissue sarcomas (STS) is limited. Response to available first line regimens is generally poor and response to second line regimens is rare. Considering the poor response of STS to available cytotoxic therapy, and the need to adequately evaluate the effectiveness of new agents, first line investigation of promising new agents is justified. Gemcitabine, a relatively new agent, has demonstrated effectiveness in several solid tumors. Limited clinical trials have suggested modest activity in STS. A multi-institutional study of gemcitabine in patients with STS, without prior chemotherapy for metastatic disease, was initiated in the Southwest Oncology Group May 1, 1998 and completed March 15, 1999. MATERIALS AND METHODS: Patients were required to have metastatic or unresectable STS with no prior chemotherapy for metastatic disease. Gastrointestinal leiomyosarcomas and stromal tumors were not eligible. Patients were required to have a performance status of 0-2, measurable disease, adequate renal, hepatic, and hematologic function. The patients were given Gemcitabine 1000 mg/M2 iv over 30 minutes on days 1, 8, and 15, 22, 29, 36, 43, 57, 64, and 71. Dosage reduction was performed for cytopenias and/or other grade 3 or 4 toxicity. RESULTS: Forty-eight patients were registered to the study. The median age was 62 years (range, 30-80) with 21 male and 25 female patients. Two patients were ineligible (1 GI stromal tumor and 1 nerve sheath tumor). Forty-six patients are evaluable for response, toxicity, and survival. There were 2 treatment-related deaths (1 renal failure and 1 hemolytic uremic syndrome). Six additional patients experienced grade 4 toxicity (3 neutropenia, 2 dyspnea, 1 vomiting, and 1 renal failure). Three of the 46 eligible patients had a partial response (7%: 95% confidence interval 1-18%) and 8 patients had stable disease (20%). Nine patients had inadequate assessments to define response. Forty-five patients have died with a median survival of 6 months (95% confidence interval 5-10 months). CONCLUSION: Gemcitabine has minimal activity as a single agent at this dose and schedule in advanced STS. The low response rate does not justify further investigation of gemcitabine as a single agent in STS.

A Phase II trial of topotecan and gemcitabine in patients with previously treated, advanced nonsmall cell lung carcinoma.

BACKGROUND: Multiple trials have been performed to evaluate second-line clinical chemotherapy in patients with advanced nonsmall cell lung carcinoma (NSCLC). However, no single agent or combination has demonstrated superior activity. METHODS: Patients with advanced NSCLC who had already received one chemotherapeutic regimen were treated with topotecan (0.75 mg/m(2) over 30 minutes, Days 1-5) and gemcitabine (400 mg/m(2) over 30 minutes, Days 1 and 5) every 21 days. RESULTS: Of 35 patients who were treated, 4 (11%) achieved a partial responses and 8 (23%) hadstable disease for at least four courses of treatment. The response rate for patients with refractory disease (progressing during frontline chemotherapy) was 18% (3 of 17) with 18% having stable disease for at least four courses of treatment. The median survival of the entire group was 7 months (range, 1.5-44 months) and 20% (7 of 35) of patients were alive 1 year from the initiation of topotecan and gemcitabine treatment. Patients with refractory disease had a median survival of 4(1/2) months, with 6-month and 1-year survival rates of 47% and 18%, respectively. During Course 1, five patients (14%) developed Grade IV neutropenia and three patients (9%) developed Grade IV thrombocytopenia. Nonhematologic toxicity was relatively mild, with one patient developing Grade III side effects (fatigue) and eight patients (23%) developing Grade II nonhematologic side effects. CONCLUSIONS: The combination of topotecan and gemcitabine demonstrated antitumor activity with a modest side effect profile in patients with advanced, previously treated NSCLC.