Complete and durable response to immune checkpoint inhibitor in a patient with refractory and metastatic hepatoblastoma

Author(s):  
Hsin-Lien Tsai ◽  
Yi-Chen Yeh ◽  
Ting-Yen Yu ◽  
Chih-Ying Lee ◽  
Giun-Yi Hung ◽  
...  
2020 ◽  
Vol 13 (2) ◽  
pp. 911-915
Author(s):  
Karam Khaddour ◽  
Emily S. Gorell ◽  
Farrokh Dehdashti ◽  
Jean Y. Tang ◽  
George Ansstas

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.


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 ◽  
...  

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.


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

2019 ◽  
Vol 25 ◽  
pp. 256
Author(s):  
Mohammad Ansari ◽  
Ula Tarabichi ◽  
Hadoun Jabri ◽  
Qiang Nai ◽  
Anis Rehman ◽  
...  

Diabetes ◽  
2018 ◽  
Vol 67 (Supplement 1) ◽  
pp. 204-LB ◽  
Author(s):  
KARA R. MIZOKAMI-STOUT ◽  
ROMA GIANCHANDANI ◽  
MARK MACEACHERN ◽  
RAVI M. IYENGAR ◽  
SARAH YENTZ ◽  
...  

Diabetes ◽  
2020 ◽  
Vol 69 (Supplement 1) ◽  
pp. 1672-P
Author(s):  
AMANDA LEITER ◽  
EMILY CARROLL ◽  
DANIELLE C. BROOKS ◽  
JENNIFER BEN SHIMOL ◽  
ELLIOT EISENBERG ◽  
...  

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