First in-human intrathecal delivery of bevacizumab for leptomeningeal spread from recurrent glioblastoma: rationale for a dose escalation trial.

PURPOSE: To outline the dose rationale for the first in-human intrathecal delivery of bevacizumab for LMS of GBM. METHODS: A 19-year-old female patient presented to Lenox Hill Hospital following thalamic GBM recurrence. She subsequently underwent two infusions of intra-arterial BEV (NCT01269853) and experienced a period of relative disease stability until progression in 2022. One month later, MRI disclosed diffuse enhancement representative of LMS of GBM. The patient subsequently underwent five cycles of IT BEV in mid-2022 (IND 162119). Doses of 25 mg, 37.5 mg, 50 mg, 50 mg, and 37.8 mg were delivered at two-week intervals between doses 1-4. The final 37.8 mg dose was given one day following her fourth dose, given that the patient was to be discharged, traveled several hours to our center, and was tolerating therapy well. Dosage was decreased due to the short interval between the final two treatments. Shortly after IT BEV completion, she received a third dose of IA BEV. RESULTS: Our patient did not show any signs of serious adverse effects or dose limiting toxicities following any of the treatments. It is difficult to determine PFS due to the rapid progression associated with LMS of GBM and rapid timeframe of treatment. CONCLUSION: LMS continues to be a devastating progression in many types of cancer, including GBM, and novel ways to deliver therapeutics may offer patients symptomatic and therapeutic benefits.

Complete Resolution of Skull Base Solitary Plasmacytoma Using Proton-Beam Radiotherapy: A Case Report.

Cranial solitary plasmacytomas are uncommon lesions, and localization to the skull base is rare. Here we present a case in a 36-year-old woman who complained of dizziness and mild headaches. Radiographic imaging indicated the presence of a solitary skull base lesion in the posterior cranial fossa. Laboratory tests and imaging excluded systemic disease. A biopsy of the lesion confirmed the diagnosis of plasmacytoma. The patient was treated with proton-beam radiation and had a complete clinical and radiographic resolution, demonstrating the previously unreported utility of monotherapy with proton-beam radiation in such cases.

Developing an emergency department order set to treat acute pain in sickle cell disease.

STUDY OBJECTIVE: Patients with sickle cell disease (SCD) have many emergency department visits because of painful vaso-occlusive episodes (VOE). Guidelines recommend treatment within 30 minutes of triage, but this is rarely achieved in clinical practice. Our goal was to develop an order set that is being implemented in the ED to facilitate and standardize emergency care for SCD patients in acute pain from VOEs presenting to the emergency department (ED) in New York City (NYC). METHODS: Using a RAND/University of California, Los Angeles modified Delphi panel, we convened a multidisciplinary panel and reviewed evidence on how to best manage SCD pain in the ED. Panelists collaboratively developed then rated 202 items that could be included in an ED order set. RESULTS: A consensus order set, a practical how-to guide for managing SCD pain in the ED, was developed based on items that received high median ratings. CONCLUSIONS: The management of acute pain experienced during VOEs is critical to patients with SCD; ED order sets, such as this one, can help standardize pain management, including at triage, evaluation, discharge, and follow-up care. After implementation in NYC EDs, studies to examine changes in quality care metrics (eg, wait times, readmissions) are planned.

A case of Moyamoya disease complicated by aHUS in a patient with a history of marginal zone B-cell lymphoma: lightning strikes thrice?

Atypical haemolytic uraemic syndrome (aHUS) is a rare, acquired thrombotic microangiopathy, mediated by complement activation, in very sick patients. Moyamoya is similarly a rare disease in which stenosis or occlusion of segment(s) of the anterior cerebral circulation leads to the formation of many thin collaterals. Other reports have described an association between HUS and Moyamoya disease in the paediatric population. However, this case study presents the exceptionally rare presentation of an adult with aHUS and Moyamoya disease in a patient who was treated with rituximab for marginal zone B-cell lymphoma.

A 30-Year-Old Carrier of Gaucher Disease with Multiple Myeloma.

We are reporting a case of a 30-year-old male with no past medical history who presented with new onset of renal failure, anemia, and splenomegaly and was diagnosed with multiple myeloma. Given the splenomegaly and the patient's Jewish heritage, blood tests were done and the patient was found to be a Gaucher disease carrier. The association of Gaucher disease and multiple myeloma has previously been reported; however, we want to describe the case of a young Gaucher disease carrier who developed multiple myeloma and provide a review of the literature.

A case of congenital prothrombin deficiency and idiopathic thrombocytopenic purpura in a pregnant female.

Hereditary prothrombin deficiency is an autosomal recessive disorder with an estimated incidence of 1 in 2 million . Presentation of the disease is variable; however, it is usually associated with moderate to severe bleeding tendencies including muscle hematomas, hemarthrosis, intracranial, mucosal, and postoperative bleeding. Here we report a case of a 35-year-old pregnant woman with congenital hypoprothrombinemia and idiopathic thrombocytopenic purpura, review the literature, and discuss its epidemiology, presentation, diagnosis, and treatment.

Unrecognized thyroid dysfunction in patients with cancer.

Abnormal thyroid function can cause subtle medical symptoms in "healthy" individuals. Because cancer can cause similar symptoms, physicians may fail to suspect and test for thyroid abnormalities. We measured thyroid function in 158 patients with various types of cancers being followed at a comprehensive cancer center and found that 16% of them had a thyroid abnormality, which had not been diagnosed and treated in any of them. We conclude that thyroid dysfunction is usually unrecognized in cancer patients and may possibly contribute to their morbidity.