Significance of emerging clinical oncology endpoints in support of overall survival

Despite a better understanding of the pathophysiology and development of newer therapeutic options, cancer remains an area with several unmet needs. Although overall survival (OS) remains a gold standard endpoint for all cancer therapies, it poses challenges such as the requirement of a long?term follow?up, a higher number of patients, and a higher financial burden. Therefore, surrogate endpoints such as progression?free survival, time to progression, duration of response, and objective response rate are being investigated and used in oncology studies. Patient?related outcomes that measure the patient抯 overall health, quality of life, and satisfaction in the long term are crucial surrogate endpoints considered for drug approval. Surrogate endpoints shorten oncology clinical studies and accelerate the evaluation and implementation of newer therapies. Emerging surrogate endpoints such as biomarkers, immune?related response criteria, minimal residual disease, and pathological complete response are increasingly being considered in oncology trials. Validation of surrogate endpoints enables their substitution for OS and gain market approval. The selection of surrogate endpoints for an oncology trial depends on cancer type and stage, the purpose of treatment, and expected duration of survival for the relevant disease. With the advent of individualized approach and complex study designs, the field of oncology is currently undergoing a paradigm shift. The use of newer surrogate endpoints will aid in accelerating the drug development process, making patient care for oncology more accessible

Neurofibromatosis type-1 with seizures and cerebrovascular malformation: a case study

Neurofibromatosis type 1 (NF1) is the most common autosomal dominant neurocutaneous among humans. Epilepsy is more prevalent in NF1 patients than in the general population. NF1 vasculopathy is also a significant but underrecognized complication of the disease, affecting both arterial and venous blood vessels. Herein, we report a 2 year old female child with seizures and multiple cafe-au-lait spots on the body. The patient was diagnosed with NF1 based on clinical findings and family history. MRI Brain revealed middle cerebral artery dysplasia. Here we discuss diagnostic and treatment challenges and briefly reviews the existing literature.

Tuberculosis of patella in an immunocompetent patient: A case report.

Isolated involvement of patella as extra-pulmonary tuberculosis is very uncommon. It is the clinical presentation of the patellar osteomyelitis that makes it prone to delay in diagnosis as reported in literature. Imaging studies, especially MRI, are of great interest for the diagnosis and to assess the extent of tuberculosis. A tissue or bacterial diagnosis is essential. Early institution of anti-tuberculous drugs minimizes the spread of the diseases, decreases the patient’s morbidity and gives a better knee functions. One rare case of patellar tuberculosis is discussed here with its clinical and radiological features.

Primary cardiac tumors: surgical experience and follow-up.

BACKGROUND: Primary cardiac tumors are rare. There are only a few reports of such tumors from India. METHODS AND RESULTS: We report our experience with 34 patients with primary cardiac tumors operated on at our institute between December 1989 and October 2001. The study group comprised 16 males and 18 females with a mean age of 40.05 +/- 13.06 years (range 7-65 years). The predominant symptoms were breathlessness and congestive heart failure. In addition, 1 patient presented with peripheral embolism with impending limb ischemia that necessitated emergency embolectomy. Echocardiography was confirmatory in the diagnosis of all the benign tumors, whereas the malignant tumors were incidentally found during surgery. All the patients survived the operation. Complete resection of the tumor was possible only in benign tumors; however, malignant tumors were partly removed to relieve obstruction. All the excised benign tumors showed no recurrence on a mean follow-up of 54.78 +/- 31.30 months (range 3-108 months). Myxoma was found in 31 patients, left ventricular fibroma in 1, and leiomyosarcoma in 2. Both the patients with malignant tumors developed recurrence postoperatively, and succumbed to extensive distant metastases. CONCLUSIONS: The long-term outcome of surgery was excellent in patients with benign cardiac tumors. However, malignant tumors had an unchanged prognosis, although symptomatic relief was achieved.