tbuckley/cabot-search huggingface dataset:
Access and Safety—The FDA Drug Approval Conundrum
Authors: Chester B. Good, Samuel K. Peasah
Journal: JAMA Network Open (2024)

William B. Feldman, MD, DPhil, MPH; Mufaddal Mahesri, MD, MPH; Ameet Sarpatwari, PhD, JD; Krista F. Huybrechts, MS, PhD; Yanmin Zhu, PhD; Catherine S. Hwang, MD, MSPH; Joyce Lii, MS, MA; Su Been Lee, BA; Sushama Kattinakere Sreedhara, MBBS, MSPH; Gita A. Toyserkani, PharmD, MBA; Esther H. Zhou, MD, PhD; Laura Zendel, PharmD; Cynthia LaCivita, PharmD; Claudia Manzo, PharmD; Gerald J. Dal Pan, MD, MHS; Aaron S. Kesselheim, MD, JD, MPH; Katsiaryna Bykov, PharmD, ScD
Lymphatic-localized Treg-mregDC crosstalk limits antigen trafficking and restrains anti-tumor immunity
Authors: Siyuan You, Shuqin Li, Lingsu Zeng, Jinsheng Song, Zifeng Li, Weiyun Li, Hengxiao Ni, Xu Xiao, Wenbo Deng, Hongye Li, Wenbo Lin, Chenyu Liang, Yanfei Zheng, Shih-Chin Cheng, Nengming Xiao, Mengsha Tong, Rongshan Yu, Jialiang Huang, Hongling Huang, Hongzhi Xu, Jiahuai Han, Jianlin Ren, Kairui Mao
Journal: Cancer Cell (2024)
Immunotherapy vs Best Supportive Care for Patients With Hepatocellular Cancer With Child-Pugh B Dysfunction
Authors: Claudia Angela Maria Fulgenzi, Bernhard Scheiner, Antonio D’Alessio, Aman Mehan, Giulia F. Manfredi, Ciro Celsa, Naoshi Nishida, Celina Ang, Thomas U. Marron, Linda Wu, Anwaar Saeed, Brooke Wietharn, Antonella Cammarota, Tiziana Pressiani, Matthias Pinter, Rohini Sharma, Jaekyung Cheon, Yi-Hsiang Huang, Pei-Chang Lee, Samuel Phen, Anuhya Gampa, Anjana Pillai, Andrea Napolitano, Caterina Vivaldi, Francesca Salani, Gianluca Masi, Marianna Silletta, Federica Lo Prinzi, Emanuela Di Giacomo, Bruno Vincenzi, Dominik Bettinger, Robert Thimme, Arndt Vogel, Martin Schönlein, Johann von Felden, Kornelius Schulze, Henning Wege, Peter R. Galle, Mario Pirisi, Joong-Won Park, Masatoshi Kudo, Lorenza Rimassa, Amit G. Singal, Paul El Tomb, Susanna Ulahannan, Alessandro Parisi, Hong Jae Chon, Wei-Fan Hsu, Giorgia Ghittoni, Calogero Cammà, Benedetta Stefanini, Franco Trevisani, Edoardo G. Giannini, Alessio Cortellini, David James Pinato
Journal: JAMA Oncology (2024)

Whether patients with Child-Pugh class B (CP-B) cancer with unresectable hepatocellular carcinoma (uHCC) benefit from active anticancer treatment vs best supportive care (BSC) is debated.
Mammalian SWI/SNF complex activity regulates POU2F3 and constitutes a targetable dependency in small cell lung cancer
Authors: Leslie Duplaquet, Kevin So, Alexander W. Ying, Shreoshi Pal Choudhuri, Xinyue Li, Grace D. Xu, Yixiang Li, Xintao Qiu, Rong Li, Shilpa Singh, Xiaoli S. Wu, Seth Hamilton, Victor D. Chien, Qi Liu, Jun Qi, Tim D.D. Somerville, Hillary M. Heiling, Emanuele Mazzola, Yenarae Lee, Thomas Zoller, Christopher R. Vakoc, John G. Doench, William C. Forrester, Tinya Abrams, Henry W. Long, Matthew J. Niederst, Benjamin J. Drapkin, Cigall Kadoch, Matthew G. Oser
Journal: Cancer Cell (2024)

Small cell lung cancers (SCLCs) are composed of heterogeneous subtypes marked by lineage-specific transcription factors, including ASCL1, NEUROD1, and POU2F3. POU2F3-positive SCLCs, ∼12% of all cases, are uniquely dependent on POU2F3 itself; as such, approaches to attenuate POU2F3 expression may represent new therapeutic opportunities. Here using genome-scale screens for regulators of POU2F3 expression and SCLC proliferation, we define mSWI/SNF complexes as top dependencies specific to POU2F3-positive SCLC. Notably, chemical disruption of mSWI/SNF ATPase activity attenuates proliferation of all POU2F3-positive SCLCs, while disruption of non-canonical BAF (ncBAF) via BRD9 degradation is effective in pure non-neuroendocrine POU2F3-SCLCs. mSWI/SNF targets to and maintains accessibility over gene loci central to POU2F3-mediated gene regulatory networks. Finally, clinical-grade pharmacologic disruption of SMARCA4/2 ATPases and BRD9 decreases POU2F3-SCLC tumor growth and increases survival in vivo. These results demonstrate mSWI/SNF-mediated governance of the POU2F3 oncogenic program and suggest mSWI/SNF inhibition as a therapeutic strategy for POU2F3-positive SCLCs.
Toward a Comprehensive Measure of Drug-Attributable Harm
Authors: Vaughan W. Rees, Connor Kubeisy, Howard K. Koh
Journal: JAMA (2024)

This Viewpoint from authors at the Harvard T.H. Chan School of Public Health summarizes approaches to addressing substance use in the US; challenges posed by metrics such as overdose deaths; and more informative metrics, such as quality-adjusted life-years, to promote health equity and improve length and quality of life and health outcomes at all levels.
Trends in long term vaping among adults in England, 2013-23: population based study
Authors: Sarah E Jackson, Harry Tattan-Birch, Lion Shahab, Jamie Brown
Journal: BMJ (2024)

Abstract Objective To examine trends in long term (&gt;6 months) vaping among adults in England. Design Population based study. Setting England. Participants 179 725 adults (≥18 years) surveyed between October 2013 and October 2023. Main outcome measures Time trends in prevalence of long term vaping using logistic regression, overall and by vaping frequency (daily or non-daily), and main type of device used (disposable, refillable, or pod). Results The proportion of adults reporting long term vaping increased non-linearly, from 1.3% (95% confidence interval 1.1% to 1.5%) in October 2013 to 10.0% (9.2% to 10.9%) in October 2023, with a particularly pronounced rise from 2021. This rise included an increase in long term daily vaping, from 0.6% (0.5% to 0.8%) to 6.7% (6.0% to 7.4%). Absolute increases in long term vaping were larger among people with a history of regular smoking (current smokers: 4.8% (4.0% to 5.8%) to 23.1% (20.4% to 25.9%); recent former smokers: 5.7% (3.4% to 9.2%) to 36.1% (27.6% to 45.4%); long term former smokers: 1.4% (1.0% to 1.9%) to 16.2% (14.2% to 18.4%)), but an increase also occurred among people who had never regularly smoked (0.1% (0.0% to 0.2%) to 3.0% (2.3% to 3.8%)). Growth was also more pronounced in young adults (eg, reaching 22.7% (19.2% to 26.5%) of 18 year olds v 4.3% (3.6% to 5.2%) of 65 year olds), including among those who had never regularly smoked (reaching 16.1% (11.1% to 22.7%) of 18 year olds v 0.3% (0.1% to 0.6%) of 65 year olds). Between October 2013 and March 2021, most long term vapers mainly or exclusively used refillable electronic cigarettes (2.5% to 3.3% of adults) and few (0.1% of adults) used disposable devices. However, prevalence of long term vaping using disposable devices subsequently rose rapidly, and by October 2023 similar proportions of adults mainly or exclusively used disposable and refillable devices (4.9% (4.2% to 5.7%) and 4.6% (4.0% to 5.3%), respectively). Conclusions The prevalence of long term vaping increased substantially among adults in England during 2013-23. Much of this increase occurred from 2021, coinciding with the rise in popularity of disposable e-cigarettes. Half of long term vapers now mainly or exclusively use disposable devices. The growth was concentrated among people with a history of regular smoking, but an increase also occurred among people who never regularly smoked, especially young adults.
Naphthalene Diimide‐Based Polycyclic Conjugated Molecule Composite CoFe<sub>2</sub>O<sub>4</sub> Nanohybrids for Photoacoustic Imaging‐Mediated Photo‐/Sonic Therapy
Authors: Gongning Chen, Fu Yang, Danping Wu, Jun Yang, Xiaojuan Hu, Longcai Liu, Yehui Kang, Weijiao Fan, Xiaozhou Mou, Yu Cai, Chunjie Hou
Journal: Advanced Healthcare Materials (2024)

Abstract The complex and harsh tumor microenvironment imped the efficacy of single‐modality tumor therapy. With the advantages of biosafety, organic/inorganic nanohybrids have attracted more and more interest of researchers, and it is critical to investigate the development of highly efficient nanohybrids for multimodality combination therapy of cancers. Herein, a naphthalene diimide‐based polycyclic conjugated molecule (NDI‐S) is designed and synthesized, which has broader light absorption in the near infrared (NIR) region, outstanding photothermal conversion ability, and excellent photostability. Inorganic CoFe 2 O 4 is synthesized via a solvothermal technique, which can produce much more reactive oxygen species (ROS) as a sonosensitizer when activated by ultrasonic (US). NDI‐S and CoFe 2 O 4 are then nanoprecipitated to create the organic/inorganic nanohybrids, NDI‐S@CoFe 2 O 4. According to the results of in vitro and in vivo experiments, NDI‐S@CoFe 2 O 4 can serve as a multifunctional nanoplatform for multimodal treatment of tumors in combination with photothermal/photodynamic/sonodynamic‐ therapy under the guidance of photoacoustic imaging, which provides a new vision of the development of organic/inorganic nanohybrids for cancer theranostics.
<i>Notes from the Field</i>: Schedule I Substances Identified in Nootropic Gummies Containing <i>Amanita muscaria</i> or Other Mushrooms — Charlottesville, Virginia, 2023–2024
Authors: Avery Michienzi, Jeremy Hamlin, Rita Farah, Lindsay Bazydlo
Journal: MMWR Morbidity and Mortality Weekly Report (2024)

Since late 2023, according to conversations between investigators and smoke shop employees, gas stations and smoke shops in Virginia have been selling mushroom gummies, marketed as nootropics (substances taken to enhance cognitive function) or psychedelics.These gummies are labeled to contain either Amanita muscaria or proprietary mushroom nootropic blends.Unlike other hallucinogenic mushrooms that contain the Drug Enforcement Administration (DEA) schedule I substance psilocybin, A. muscaria is currently legal.* A. muscaria contains ibotenic acid and muscimol and is used less commonly as a hallucinogen than are psilocybin-containing mushrooms because A. muscaria can cause undesired symptoms, including gastrointestinal upset, agitation, and seizures (1-3).During September 2023-June 2024, five patients, including one child, were evaluated in Virginia after ingestion of gummies listing A. muscaria as an ingredient.Because the actual contents of these novel mushroom gummies are unknown, samples from five brands were obtained for testing using liquid chromatography-mass spectrometry.
Upconverting Ultra‐Thin Bi<sub>2</sub>O<sub>2</sub>CO<sub>3</sub> Nanosheets for Synergistic Photodynamic Therapy and Radiotherapy
Authors: Shuzhi Shi, Cheng Liao, Yufu Liu, Jinming Liu, Jinliang Liu, Yi Zhang, Yong Zhang, Qingsong Mei
Journal: Advanced Healthcare Materials (2024)

Synergistic therapy has become the major therapeutic method for malignant tumors in clinical. Photodynamic therapy (PDT) and radiotherapy (RT) always combine together because of their identical anti-tumor mechanisms, that is reactive oxygen species are generated by the use of radiosensitizers after irradiation by X-ray to efficiently kill cancer cells, PDT also follows similar mechanism. Full exposure of energy-absorbing species in nanomaterials to X-ray or near-infrared light irradiation makes the energy interchange between nanomaterials and surrounding H
Childhood Lead Exposure Linked to Apple Cinnamon Fruit Puree Pouches — North Carolina, June 2023–January 2024
Authors: Melanie D. Napier, Alan Huneycutt, Carissa Moore, Chris Goforth, Marc Komlos, Veronica Bryant, Scott M. Shone, Larry D. Michael, Edward H. Norman
Journal: MMWR Morbidity and Mortality Weekly Report (2024)

Lead exposure is toxic even at low levels, resulting in impairments that can affect a child's lifelong success.In North Carolina, testing for lead is encouraged for all children at ages 1 and 2 years and required for children covered by Medicaid; investigations are performed to identify potential exposure sources for children with blood lead levels (BLLs) ≥5 μg/dL.During June-August 2023, routine lead testing identified four asymptomatic North Carolina children with BLLs ≥5 μg/dL.Home investigations identified only WanaBana brand apple cinnamon fruit puree pouches as a potential exposure source; product samples contained 1.9-3.0ppm of lead.An expanded nationwide investigation led to identification of approximately 500 cases of childhood lead exposure believed to be linked to consumption of apple cinnamon purees, including 22 cases in North Carolina.Fewer than one half (45%) of the 22 North Carolina cases were among children covered by Medicaid.A coordinated multiagency communication strategy was implemented in North Carolina to notify consumers of the hazard and provide recommendations for preventing further exposure.The Food and Drug Administration issued a nationwide public health advisory on October 28, 2023; 2 days later, the manufacturer issued a voluntary recall.Routine testing of young children for lead exposure, combined with thorough environmental investigations, can identify emerging sources of lead exposure and limit further harm.
The SCRUM-MONSTAR Cancer-Omics Ecosystem: Striving for a Quantum Leap in Precision Medicine
Authors: Tadayoshi Hashimoto, Yoshiaki Nakamura, Takao Fujisawa, Mitsuho Imai, Taro Shibuki, Naoko Iida, Hiroshi Ozaki, Norio Nonomura, Chigusa Morizane, Hiroji Iwata, Susumu Okano, Wataru Yamagami, Naoya Yamazaki, Shigenori Kadowaki, Hiroya Taniguchi, Makoto Ueno, Shogen Boku, Eiji Oki, Yoshito Komatsu, Satoshi Yuki, Akitaka Makiyama, Tomoyuki Otsuka, Hiroki Hara, Naohiro Okano, Tomohiro Nishina, Yasutoshi Sakamoto, Izumi Miki, Shin Kobayashi, Junichiro Yuda, Shun-Ichiro Kageyama, Michiko Nagamine, Shingo Sakashita, Naoya Sakamoto, Riu Yamashita, Yoshikatsu Koga, Hideaki Bando, Genichiro Ishii, Takeshi Kuwata, Woong-Yang Park, Atsushi Ohtsu, Takayuki Yoshino
Journal: Cancer Discovery (2024)

Abstract The SCRUM-Japan MONSTAR-SCREEN consortium is a nationwide molecular profiling project employing artificial intelligence–driven multiomics analyses for patients with advanced malignancies, aiming to develop novel therapeutics and diagnostics and deliver effective drugs to patients. Concurrently, studies assessing molecular residual disease–based precision medicine for resectable solid tumors, including CIRCULATE-Japan, are ongoing. The substantial data generated by these platforms are stored within a state-of-the-art supercomputing infrastructure, VAPOR CONE. Since 2015, our project has registered over 24,000 patients as of December 2023. Among 16,144 patients with advanced solid tumors enrolled in MONSTAR-SCREEN projects, 5.0% have participated in matched clinical trials, demonstrating a 29.2% objective response rate and 14.8-month median survival (95% CI, 13.4–16.3) for patients treated in the matched clinical trials. Notably, patients who received matched therapy demonstrated significantly prolonged overall survival compared with those who did not (hazard ratio 0.77; 95% confidence interval, 0.71–0.83). Significance: Our nationwide molecular profiling initiative played pivotal roles in facilitating the enrollment of patients with advanced solid tumors into matched clinical trials and highlighted the substantial survival benefits of patients treated with matched therapy. We aim to facilitate an industry–academia data-sharing infrastructure ecosystem, fostering new drug discovery paradigms and precision medicine.
Progress Toward Achieving and Sustaining Maternal and Neonatal Tetanus Elimination — Worldwide, 2000–2022
Authors: Camille E. Jones, Nasir Yusuf, Bilal Ahmed, Modibo Kassogue, Annemarie Wasley, Florence A. Kanu
Journal: MMWR Morbidity and Mortality Weekly Report (2024)

Tetanus remains a considerable cause of mortality among undervaccinated mothers and their infants following unhygienic deliveries, especially in low-income countries.Strategies of the maternal and neonatal tetanus elimination (MNTE) initiative, which targets 59 priority countries, include strengthening antenatal immunization of pregnant women with tetanus toxoid-containing vaccines (TTCVs); conducting TTCV supplementary immunization activities among women of reproductive age in high-risk districts; optimizing access to skilled birth attendants to ensure clean deliveries and umbilical cord care practices; and identifying and investigating suspected neonatal tetanus cases.This report updates a previous report and describes progress toward MNTE during 2000-2022.By December 2022, 47 (80%) of 59 priority countries were validated to have achieved MNTE.In 2022, among the 50 countries that reported coverage with ≥2 doses of TTCV among pregnant women, 16 (32%) reported coverage of ≥80%.In 2022, among 47 validated countries, 26 (55%) reported that ≥70% of births were assisted by skilled birth attendants.Reported neonatal tetanus cases worldwide decreased 89%, from 17,935 in 2000 to 1,995 in 2021; estimated neonatal tetanus deaths decreased 84%, from 46,898 to 7,719.However, the global disruption of routine immunization caused by the COVID-19 pandemic impeded MNTE progress.Since 2020, reported neonatal tetanus cases have increased in 18 (31%) priority countries.Integration of MNTE strategies into priority countries' national postpandemic immunization recovery activities is needed to achieve and sustain global elimination.
SARS-CoV-2 brainstem encephalitis in human inherited DBR1 deficiency
Authors: Yi-Hao Chan, Vanja Lundberg, Jérémie Le Pen, Jiayi Yuan, Danyel Lee, Francesca Pinci, Stefano Volpi, Koji Nakajima, Vincent Bondet, Sanna Åkesson, Noopur V. Khobrekar, Aaron Bodansky, Likun Du, Tina Melander, Alice-Andrée Mariaggi, Yoann Seeleuthner, Tariq Shikh Saleh, Debanjana Chakravarty, Per Marits, Kerry Dobbs, Sofie Vonlanthen, Viktoria Hennings, Karolina Thörn, Darawan Rinchai, Lucy Bizien, Matthieu Chaldebas, Ali Sobh, Tayfun Özçelik, Sevgi Keles, Suzan A. AlKhater, Carolina Prando, Isabelle Meyts, Paul Bastard, Alessandro Borghesi, Aziz Bousfiha, Oksana Boyarchuk, Petter Brodin, Jacinta Bustamante, Giorgio Casari, Rémi Chevalier, John Christodoulou, Roger Colobran, Antonio Condino-Neto, Juan Carlos Aldave Becerra, Lisa Arkin, Evangelos Andreakos, Christian W. Thorball, Sara Espinosa, Carlos Flores, Amyrath Geraldo, Rabih Halwani, Nevin Hatipoğlu, Brahim Melaiki, Jacques Fellay, Alenka Gagro, Yuval Itan, Chandima Jeewandara, Filomeen Haerynck, Davood Mansouri, Leslie Naesens, Lisa F.P. Ng, Keisuke Okamoto, Pere Soler-Palacin, Laurent Renia, Aurora Pujol Onofre, Igor B. Resnick, José Luis Franco Restrepo, Jacques G. Rivière, Anna Scherbina, Anna Šedivá, Mikko R.J. Seppänen, Helen Su, Stuart G Tangye, Sehime G. Temel, Ahmad Abou Tayoun, Stuart Turvey, K.M. Furkan Uddin, Diederik van de Beek, Tom Le Voyer, Donald C. Vinh, Shen-Ying Zhang, Jean-Laurent Casanova, Michael R. Wilson, Jérémie Rosain, Emmanuelle Jouanguy, Mélodie Aubart, Laurent Abel, Trine H. Mogensen, Qiang Pan-Hammarström, Daxing Gao, Darragh Duffy, Aurélie Cobat, Stefan Berg, Luigi D. Notarangelo, Oliver Harschnitz, Charles M. Rice, Lorenz Studer, Jean-Laurent Casanova, Olov Ekwall, Shen-Ying Zhang
Journal: The Journal of Experimental Medicine (2024)

Inherited deficiency of the RNA lariat–debranching enzyme 1 (DBR1) is a rare etiology of brainstem viral encephalitis. The cellular basis of disease and the range of viral predisposition are unclear. We report inherited DBR1 deficiency in a 14-year-old boy who suffered from isolated SARS-CoV-2 brainstem encephalitis. The patient is homozygous for a previously reported hypomorphic and pathogenic DBR1 variant (I120T). Consistently, DBR1 I120T/I120T fibroblasts from affected individuals from this and another unrelated kindred have similarly low levels of DBR1 protein and high levels of RNA lariats. DBR1 I120T/I120T human pluripotent stem cell (hPSC)–derived hindbrain neurons are highly susceptible to SARS-CoV-2 infection. Exogenous WT DBR1 expression in DBR1 I120T/I120T fibroblasts and hindbrain neurons rescued the RNA lariat accumulation phenotype. Moreover, expression of exogenous RNA lariats, mimicking DBR1 deficiency, increased the susceptibility of WT hindbrain neurons to SARS-CoV-2 infection. Inborn errors of DBR1 impair hindbrain neuron–intrinsic antiviral immunity, predisposing to viral infections of the brainstem, including that by SARS-CoV-2.
Safety and efficacy of <scp>aficamten</scp> in patients with non‐obstructive hypertrophic cardiomyopathy: A 36‐week analysis from <scp>FOREST</scp>‐<scp>HCM</scp>
Authors: Ahmad Masri, Roberto Barriales‐Villa, Perry Elliott, Michael E. Nassif, Artur Oreziak, Anjali T. Owens, Albree Tower‐Rader, Stephen B. Heitner, Stuart Kupfer, Fady I. Malik, Chiara Melloni, Lisa Meng, Jenny Wei, Sara Saberi
Journal: European Journal of Heart Failure (2024)

The aim of this study was to report safety and efficacy of aficamten in patients with non-obstructive hypertrophic cardiomyopathy (nHCM) over 36 weeks in the ongoing FOREST-HCM trial.
Risk of myeloproliferative neoplasms among <scp>U.S.</scp> Veterans from Korean, Vietnam, and Persian Gulf War eras
Authors: Andrew Tiu, Zoe McKinnell, Shanshan Liu, Puneet Gill, Martha Antonio, Zoe Shancer, Nandan Srinivasa, Guoqing Diao, Ramesh Subrahmanyam, Craig M. Kessler, Maneesh Jain
Journal: American Journal of Hematology (2024)

Abstract The Promise to Address Comprehensive Toxics (PACT) Act expanded U.S. Veterans' health care and benefits for conditions linked to service‐connected exposures (e.g., Burn Pits, Agent Orange). However, myeloproliferative neoplasms (MPN) are not recognized as presumptive conditions for Veterans exposed to these toxic substances. This study evaluated the development of MPN among U.S. Veterans from the Korean, Vietnam, and Persian Gulf War eras. This retrospective cohort study included 65 425 Korean War era Veterans; 211 927 Vietnam War era Veterans; and 214 007 Persian Gulf War era Veterans from January 1, 2006, to January 26, 2023. Veterans with MPN, thrombosis, bleeding, and cardiovascular risk factors were identified through ICD‐9 and ‐10 codes. Veterans from the Persian Gulf War era had the highest risk of developing MPN compared with Veterans from the Korean and Vietnam War eras, hazard ratio (HR) 4.92, 95% confidence interval (CI) 4.20–5.75 and HR 2.49, 95% CI 2.20–2.82, both p &lt; .0001, respectively. Vietnam War era Veterans also had a higher risk of MPN development compared with Korean War era Veterans, HR 1.97, 95% CI 1.77–2.21, p &lt; .0001. Persian Gulf War era Veterans were diagnosed with MPN at an earlier age, had higher risks of thrombosis and bleeding, and had lower survival rates compared with Korean War and Vietnam War era Veterans. This study reinforces evidence that environmental and occupational hazards increase the risk of clonal myeloid disorders and related complications, impacting overall survival with MPN. Limitations include the inability to confirm clonality and fully verify deployment and exposure status.
Screening adolescent transgender-related distress: Gender preoccupation and stability questionnaire demonstrates excellent criterion validity with multi-disciplinary, pediatric gender specialist assessment
Authors: Olivia J. E. Donaghy, Vanessa Cobham, Ashleigh Lin
Journal: International Journal of Transgender Health (2024)

Objective To assess the psychometric properties of a gender-related distress questionnaire in adolescents questioning or experiencing gender incongruence and/or gender dysphoria, aged under 18 years, with multi-disciplinary, specialist pediatric gender assessment.Design & Setting: Prospective cohort study at state-wide tertiary children's hospital gender clinic (2017–2021).