scholarly journals Targeted Therapy with Sirolimus and Nivolumab in a Child with Refractory Multifocal Anaplastic Ependymoma

Reports ◽  
2021 ◽  
Vol 4 (2) ◽  
pp. 12
Author(s):  
Katia Perruccio ◽  
Angela Mastronuzzi ◽  
Marco Lupattelli ◽  
Francesco Arcioni ◽  
Ilaria Capolsini ◽  
...  
Keyword(s):  
Immune Checkpoint Inhibitor ◽  
Checkpoint Inhibitor ◽  
Mammalian Target Of Rapamycin ◽  
Experimental Therapy ◽  
Anaplastic Ependymoma ◽  
Durable Response ◽  
The Third ◽  
Pediatric Ependymoma ◽  
Relapse Tumor ◽  
Therapeutical Options

Pediatric ependymoma (EPN) is the third most common central nervous system (CNS) tumor, with 90% arising intracranially. Management typically involves maximal surgical resection and radiotherapy, but patients’ outcome is poor. Moreover, there are only a few therapeutical options available for recurrent or refractory disease. In this report, we present the case of a 7-year-old girl with relapsed refractory multifocal grade III EPN who failed conventional treatments and experienced a stable and durable response to the immune checkpoint inhibitor (ICPI) nivolumab in association with the mammalian target of rapamycin (m-TOR) inhibitor sirolimus. This experimental therapy was targeted on immune phenotypical analyses of the patient’s last relapse tumor sample, and this procedure should be routinely done to find new possible therapeutical approaches in recurrent solid tumors.

scholarly journals Case of nivolumab-induced sclerosing cholangitis: lessons from long-term follow-up

2020 ◽  
Vol 7 (1) ◽  
pp. e000487
Author(s):  
Tess McClure ◽  
Wanyuan Cui ◽  
Khashayar Asadi ◽  
Thomas John ◽  
Adam Testro
Keyword(s):  
Sclerosing Cholangitis ◽  
Immune Checkpoint Inhibitor ◽  
Checkpoint Inhibitor ◽  
Solid Organ ◽  
Long Term Outcomes ◽  
Durable Response ◽  
Long Term Follow Up ◽  
Proven Case

Nivolumab is an immune checkpoint inhibitor used to treat multiple solid-organ malignancies. While many of its immune-related adverse events are well established, nivolumab-induced sclerosing cholangitis remains poorly characterised, with no defined diagnostic criteria. Moreover, data regarding long-term outcomes are particularly lacking. We present a biopsy-proven case of nivolumab-induced sclerosing cholangitis, which uniquely captures 18 months of follow-up post-treatment. Our case highlights key features of intrahepatic subtype sclerosing cholangitis and suggests durable response to corticosteroid therapy.

scholarly journals Induced Remission of Metastatic Squamous Cell Carcinoma with an Immune Checkpoint Inhibitor in a Patient with Recessive Dystrophic Epidermolysis Bullosa

2020 ◽  
Vol 13 (2) ◽  
pp. 911-915
Author(s):  
Karam Khaddour ◽  
Emily S. Gorell ◽  
Farrokh Dehdashti ◽  
Jean Y. Tang ◽  
George Ansstas
Keyword(s):  
Squamous Cell Carcinoma ◽  
Cell Carcinoma ◽  
Squamous Cell ◽  
Epidermolysis Bullosa ◽  
Immune Checkpoint ◽  
Immune Checkpoint Inhibitor ◽  
Checkpoint Inhibitor ◽  
Durable Response ◽  
Dystrophic Epidermolysis Bullosa ◽  
Recessive Dystrophic Epidermolysis Bullosa

Recessive dystrophic epidermolysis bullosa (RDEB) is a genodermatosis that leads to skin fragility and chronic wound formation. Patients with RDEB are at risk for cutaneous squamous cell carcinoma (SCC) which is a major cause of morbidity and mortality in these patients. No standard of care exists for the treatment of SCC in this patient population and therapy is based on anecdotal reports and expert opinion. We report a 32-year-old man with RDEB with previously localized SCC who later developed metastatic SCC. He was started on cemiplimab (an immune checkpoint inhibitor) 350 mg IV every 3 weeks. An objective radiological response was noted within 3 cycles. On 14 months follow-up, there was a durable response to treatment clinically and on imaging, without immune-related adverse events. To our knowledge, this is the first case report describing safe administration of immune checkpoint inhibitors in a patient with RDEB with objective and durable response of metastatic SCC. Larger case series and controlled clinical trials are needed to further investigate these medications in the RDEB population, given their high burden of aggressive and often lethal SCC.

scholarly journals Major Durable Response of Pembrolizumab in Chemotherapy Refractory Small Cell Bladder Cancer: A Case Report

2020 ◽  
Vol 13 (3) ◽  
pp. 1059-1066
Author(s):  
Olav Toai Duc Nguyen ◽  
Stein Harald Sundstrøm ◽  
Ganna Surzhykova Westvik ◽  
Ane Karoline Stræte Røttereng ◽  
Mona Røli Melhus ◽  
...  
Keyword(s):  
Lung Cancer ◽  
Bladder Cancer ◽  
Case Report ◽  
Immune Checkpoint ◽  
Immune Checkpoint Inhibitor ◽  
Checkpoint Inhibitor ◽  
Small Cell ◽  
Small Cell Lung ◽  
Durable Response ◽  
Cell Bladder Cancer

Small cell carcinoma of the urinary bladder is a rare subtype (incidence of 1–9/1,000,000), characterized by an aggressive behavior with early metastasis and poor prognosis. Chemotherapy, radiation, and surgery are the usual treatment options, but to date, no accepted standard treatment exists. Since small cell bladder cancer shares similar clinicopathological features with small cell lung cancer, the same type of chemotherapy has been used. Recently, immune checkpoint inhibitors have shown effect in small cell lung cancer, but data regarding small cell bladder cancer is insufficient. Here we present a case where a 73-year-old male with chemorefractory metastatic small cell bladder cancer received a successful treatment with immune checkpoint inhibitor pembrolizumab resulting in a major durable response and no side effects. To our knowledge, this is the second case report on successful treatment of the rare subtype of small cell bladder cancer with an immune checkpoint inhibitor, supporting the use of pembrolizumab as a therapeutic option for small cell bladder cancer. Serum neuron-specific enolase was a useful biomarker both for chemo- and immunotherapy response.

Durable response after immune checkpoint inhibitor-related diabetes in mismatch repair deficient pancreatic cancer

Immunotherapy ◽  
2021 ◽  
Author(s):  
Erica S Tsang ◽  
Evan J Walker ◽  
Julia Carnevale ◽  
George A Fisher ◽  
Andrew H Ko
Keyword(s):  
Mismatch Repair ◽  
Immune Checkpoint Inhibitor ◽  
Checkpoint Inhibitor ◽  
Locally Advanced ◽  
Protein Deficiency ◽  
Radiographic Evidence ◽  
Durable Response ◽  
Repair Protein ◽  
Discontinuation Of Treatment ◽  
Mismatch Repair Protein

Mismatch repair protein deficiency occurs in 0.8–2% of pancreatic ductal adenocarcinomas and confers susceptibility to immunotherapy. Herein, we report the case of a patient with Lynch syndrome-associated, locally advanced mismatch repair protein deficiency pancreatic ductal adenocarcinomas who demonstrated a sustained response to second-line treatment with pembrolizumab, but eventually developed immune-related diabetic ketoacidosis requiring discontinuation of treatment. He has since remained in remission, off treatment, over the following 3 years, with regular surveillance showing no clinical or radiographic evidence of disease progression. The patient’s unusual disease course raises the question of whether this serious immune-related adverse event affecting the organ of malignant involvement may have predicted his remarkable and durable response.

759 IMMUNE CHECKPOINT INHIBITOR-ASSOCIATED CELIAC DISEASE

2020 ◽  
Vol 158 (6) ◽  
pp. S-156
Author(s):  
Yousef R. Badran ◽  
Angela Shih ◽  
Donna Leet ◽  
Alexandra Coromilas ◽  
Jonathan Chen ◽  
...  
Keyword(s):  
Celiac Disease ◽  
Immune Checkpoint ◽  
Immune Checkpoint Inhibitor ◽  
Checkpoint Inhibitor

Abstract #514 Secondary Adrenal Insufficiency Due to the Immune Checkpoint Inhibitor Pembrolizumab

2019 ◽  
Vol 25 ◽  
pp. 256
Author(s):  
Mohammad Ansari ◽  
Ula Tarabichi ◽  
Hadoun Jabri ◽  
Qiang Nai ◽  
Anis Rehman ◽  
...  
Keyword(s):  
Adrenal Insufficiency ◽  
Immune Checkpoint ◽  
Immune Checkpoint Inhibitor ◽  
Checkpoint Inhibitor ◽  
Secondary Adrenal Insufficiency
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