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Idecabtagene Vicleucel in Relapsed and Refractory Multiple Myeloma

Munshi, Nikhil C; Anderson, Larry D; Shah, Nina; Madduri, Deepu; Berdeja, Jesús; Lonial, Sagar; Raje, Noopur; Lin, Yi; Siegel, David; Oriol, Albert; Moreau, Philippe; Yakoub-Agha, Ibrahim; Delforge, Michel; Cavo, Michele; Einsele, Hermann; Goldschmidt, Hartmut; Weisel, Katja; Rambaldi, Alessandro; Reece, Donna; Petrocca, Fabio; Massaro, Monica; Connarn, Jamie N; Kaiser, Shari; Patel, Payal; Huang, Liping; Campbell, Timothy B; Hege, Kristen; San-Miguel, Jesús
BACKGROUND:Idecabtagene vicleucel (ide-cel, also called bb2121), a B-cell maturation antigen-directed chimeric antigen receptor (CAR) T-cell therapy, has shown clinical activity with expected CAR T-cell toxic effects in patients with relapsed and refractory multiple myeloma. METHODS:CAR-positive (CAR+) T cells. The primary end point was an overall response (partial response or better); a key secondary end point was a complete response or better (comprising complete and stringent complete responses). RESULTS:nucleated cells) was confirmed in 33 patients, representing 26% of all 128 patients who were treated and 79% of the 42 patients who had a complete response or better. The median progression-free survival was 8.8 months (95% confidence interval, 5.6 to 11.6). Common toxic effects among the 128 treated patients included neutropenia in 117 patients (91%), anemia in 89 (70%), and thrombocytopenia in 81 (63%). Cytokine release syndrome was reported in 107 patients (84%), including 7 (5%) who had events of grade 3 or higher. Neurotoxic effects developed in 23 patients (18%) and were of grade 3 in 4 patients (3%); no neurotoxic effects higher than grade 3 occurred. Cellular kinetic analysis confirmed CAR+ T cells in 29 of 49 patients (59%) at 6 months and 4 of 11 patients (36%) at 12 months after infusion. CONCLUSIONS:Ide-cel induced responses in a majority of heavily pretreated patients with refractory and relapsed myeloma; MRD-negative status was achieved in 26% of treated patients. Almost all patients had grade 3 or 4 toxic effects, most commonly hematologic toxic effects and cytokine release syndrome. (Funded by bluebird bio and Celgene, a Bristol-Myers Squibb company; KarMMa ClinicalTrials.gov number, NCT03361748.).
PMID: 33626253
ISSN: 1533-4406
CID: 4851372

Subconjunctival Lipid Leakage From Deep Orbital Dermoid Cysts

Vahdani, Kaveh; Godfrey, Kyle J; Patel, Payal; Rose, Geoffrey E
Orbital dermoid cysts are fairly common and arise by sequestration of surface ectoderm along skeletal suture lines during embryologic development. Although anterior dermoid cysts are generally evident in infancy, deeper cysts typically present with inflammatory symptoms in adulthood. The authors report the clinical and imaging features for 6 cases of deep orbital dermoid cysts that were identified by the presence of subconjunctival lipid droplets; to the authors' knowledge, this case report represents the largest series of dermoids with such a presentation. In the absence of prior vitreoretinal surgery, subconjunctival lipid droplets are an important clinical sign suggesting the presence of a deep orbital dermoid cyst.
PMID: 32118842
ISSN: 1537-2677
CID: 4340472

Reply re: "Facial Transplantation and Ocular Considerations" [Letter]

Greenfield, Jason A; Kantar, Rami S; Rifkin, William J; Sosin, Michael; Diaz-Siso, J Rodrigo; Patel, Payal; Fleming, James C; Iliff, Nicholas T; Lee, Bradford W; Rodriguez, Eduardo D
PMID: 32658141
ISSN: 1537-2677
CID: 4629682

Congenital respiratory-type ciliated cyst of the lacrimal sac [Letter]

Charles, Norman C; Jakobiec, Frederick A; Chong, Jillian K; Godfrey, Kyle J; Patel, Payal; Ma, Lina
PMID: 31712004
ISSN: 1715-3360
CID: 4186792

Fibrous Dysplasia-like Lacrimal Sac Tumor Associated With Dacryocystitis

Charles, Norman C; Hoda, Syed T; Patel, Payal; Kim, Eleanore T; Belinsky, Irina
A 72-year-old woman who presented with right-sided epiphora and conjunctivitis underwent a probing and irrigation procedure with normal results. She improved with antibiotic-steroid drops. A swelling in the medial canthal region completely resolved. One year later, she returned with symptoms of dacryocystitis. An external dacryocystorhinostomy was performed. Characteristic dacryoliths were removed from the sac lumen, and biopsy of the sac wall showed spicules of lamellar bone within a fibrous stroma. Diagnosed as fibrous dysplasia of the lacrimal sac, this rare entity represents the second such case in the literature.
PMID: 31895726
ISSN: 1537-2677
CID: 4251682

Solid Variant of a Proliferative Apocrine Hidrocystoma

Charles, Norman C; Patel, Payal
A cystic lesion of the eyelid margin in a 37-year-old man was diagnosed clinically as an apocrine hidrocystoma, a common lesion in that location. Histopathologic examination confirmed the presence of a hidrocystoma but also disclosed an extensive proliferation of bland epithelial cells arising from the inner layer of the cyst. The authors further characterized this unique occurrence on the spectrum of Moll gland eyelid tumors by immunohistochemical analysis.
PMID: 30893188
ISSN: 1537-2677
CID: 3735132

Ocular Considerations in Face Transplantation: Report of 2 Cases and Review of the Literature

Greenfield, Jason A; Kantar, Rami S; Rifkin, William J; Sosin, Michael; Diaz-Siso, J Rodrigo; Patel, Payal; Fleming, James C; Iliff, Nicholas T; Lee, Bradford W; Rodriguez, Eduardo D
PURPOSE/OBJECTIVE:Periorbital injuries are common in face transplantation (FT) candidates. It is therefore essential that the ophthalmologist play a central role in the multidisciplinary treatment of these patients. In this study, the authors perform a comprehensive review of all procedures involving periorbital components, provide an update for the ophthalmology community regarding the current state of the field, and present 2 cases. METHODS:A comprehensive review of the literature for all FT procedures including periorbital components was performed. The authors also present 2 patients who received FT including periorbital components for extensive facial disfigurement. One patient sustained high-energy avulsive ballistic injury and underwent a total face, double jaw, and tongue transplant in 2012. The second patient received a total face, eyelids, ears, and skeletal subunits transplant for extensive facial burns in 2015. RESULTS:Literature review demonstrated that 22 (54%) of the 41 patients undergoing FT received allografts containing periorbital components. Only 14 cases (64%) reported on the presence of ocular and periocular complications. The most common complications consisted of lower eyelid ectropion and lagophthalmos, and nearly all required revisional procedures. Both patients presented with significant periorbital scarring and demonstrated good visual acuity and aesthetic outcomes at postoperative follow up between 6 and 28 months. CONCLUSIONS:Face transplantation can address extensive facial and periorbital disfigurement with satisfactory functional and aesthetic outcomes. The majority of FT performed to date have included periorbital components, and postoperative ocular and periocular complications are common. It is critical for ophthalmologists to play a central role in the care of these patients.
PMID: 30550500
ISSN: 1537-2677
CID: 4153332

Staged embolization and excision of an arteriovenous malformation involving the eyelid and orbit

Tsui, Edmund; Dunbar, Kristen E; Kim, Eleanore T; Patel, Payal
PMID: 30732507
ISSN: 1744-5108
CID: 3632362

Partially Pigmented Eyelid Basal Cell Carcinoma With Unusual Trabecular Histologic Pattern

Charles, Norman C; Patel, Payal
PMID: 28704295
ISSN: 1537-2677
CID: 2630722

Nasal Glioma: A Rare Cause of Congenital Inner Canthal Swelling

Charles, Norman C; Lisman, Richard D; Patel, Payal; Callahan, Alison B
Nasal glioma, encephalocele, and ectopic brain are rare congenital anomalies. The terminology applied to these entities has been historically confusing. In many cases, the terms overlap and may be employed synonymously although some authors emphasize their differences. The authors describe herein a child with an inner canthal mass of brain-like tissue that they interpret as nasal glioma, a variety of encephalocele that has lost its connection to the intracranial contents. This research was conducted in conformity with the Helsinki Declaration and Health Insurance Portability and Accountability Act regulations.
PMID: 29505469
ISSN: 1537-2677
CID: 2975112