Cancer
Permanent URI for this collectionhttps://hdl.handle.net/20.500.12503/21618
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Browsing Cancer by Author "Akers, Lauren"
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Item Trial of Pazopanib in a Multiply Relapsed Osteosarcoma Patient(2019-03-05) Albritton, Karen; Akers, Lauren; Ray, Anish; Basha, Riyaz; Elete, KunalBackground: Osteosarcomas (OS) are typically found among adolescents and young adults and usually affect the long bones around the knee. The current treatment options for relapsed OS include surgery, chemotherapy, targeted therapy, or some combination of these modalities. Constitutive activation of tyrosine kinase mediated pathways leading to up-regulation of cell division and growth have been implicated in OS. This study identifies a patient at Cook Children’s Medical Center (CCMC) who, upon relapse, was treated with pazopanib, a multi-tyrosine kinase inhibitor, on compassionate basis, which led to stability of disease, along with treatment related toxicities. Case Information: A 25-year-old female initially presented with left femur osteosarcoma and was treated with chemotherapy consisting of methotrexate, doxorubicin and cisplatin followed by amputation and neo-adjuvant chemotherapy. Following 6 years of remission, she presented with two lung masses that were resected along with chemotherapy and radiation. After almost 2.5 years, she presented with progressive tumor in the right pleural base for which she was treated with pazopanib. She demonstrated positive response with stable size of tumor and increased homogeneity (suggestive of tumor necrosis) but ended treatment after 3 months due to hypothyroidism and GI toxicity, namely diarrhea. Within just 1 week of discontinuation, she had a concerning increase of 17% in her lesion. Thereafter, she has relapsed multiply but remains alive 18 months after discontinuing pazopanib. Conclusion: Despite the positive response seen to pazopanib, it’s toxicity profile can be over bearing for patients. In a retrospective analysis by Velho et al, a study involving 113 patients treated with pazopanib resulted in about 12% of those discontinuing treatment due to fatigue, diarrhea, and nausea/vomiting. In another study, described by Umeda et al, 3 patients with relapsed osteosarcoma who were treated with pazopanib were all alive at 21 months or longer. Of those, two discontinued treatment despite positive response due to nausea/fatigue, lymphopenia, anemia, hypothyroidism, and elevated alkaline phosphatase. Our experience as well of those as others suggests that pazopanib may have a role in prolonging survival among patients with osteosarcoma, however the extent of the side effects has clearly contributed to a lesser than optimal length of treatment.Item Trial of Sirolimus in Gorham Disease(2019-03-05) Heym, Kenneth; Akers, Lauren; Ray, Anish; Merchant, ZahraBackground: Gorham Disease, also known as Gorham-Stout Disease, massive osteomyelitis, or vanishing bone disease, is a rare disorder of poorly understood etiology. Patients undergo bone resorption without proliferation and have significant soft-tissue swelling. No standard treatment is currently available. Novel use of the MTOR inhibitor sirolimus has shown some promise in slowing the progression of this devastating disorder. Immunosuppressant agents such as sirolimus have been used effectively in transplant patients for prophylaxis of organ rejection. We hypothesize that this agent can also successfully slow the progression of bone resporption in patients with Gorham Disease. This case series describes the diagnosis, progression, and therapy of three patients in the Cook Children’s Medical Center hematology-oncology department with varying levels of severity of Gorham Disease. Case Information: Three patients were diagnosed with Gorham Disease between 2011 and 2014 and are now being treated within the Cook Children’s Medical Center Hematology-Oncology practice. One patient has involvement of her skull base and ear canals, diagnosed after ear canal abnormalities were detected on CT following meningitis. The second patient has involvement of her posterior ribs and T7-T12 vertebral bodies, with thoracic instability and necessity of either remaining in the supine position or wearing a back brace. The third patient has involvement of his left lower extremity and left hemipelvis, necessitating a left above knee amputation and subsequent disease progression. The first two patients have had radiographic improvement after the addition of twice daily sirolimus, while the third patient has had steadying of his disease. Conclusions: Gorham Disease is a rare condition with possibly devastating effects. The introduction of sirolimus, in select cases, has appeared to either steady or slowly reverse the progression of the disease. While more studies need to be performed to understand the full effects of sirolimus on this disease, it has the potential to have a significant role in the treatment of Gorham.