Catalogue Search | MBRL
Search Results Heading
Explore the vast range of titles available.
MBRLSearchResults
-
DisciplineDiscipline
-
Is Peer ReviewedIs Peer Reviewed
-
Item TypeItem Type
-
SubjectSubject
-
YearFrom:-To:
-
More FiltersMore FiltersSourceLanguage
Done
Filters
Reset
787,951
result(s) for
"Paediatrics"
Sort by:
Author index
in
Pediatrics
2024
Abbara Aula, 6535 Abbas Ayesha, 6256 Abbas Mohamed, 6818 Abdalla Hesham, 6577 Abdelaziz Maysara, 6618 Abdelbari Heba, 6884 Abdelhady Ammar, 5995 Abdelhafiz Khalid, 5982 Abdelrahman Dalia, 5982 Abdelreheem Mahmoud, 6018 Abdelwahab Abdellatif Hamdy, 5991 Abdurrahman Muhammad Fahd, 6730 Abelian Artur, 6645, 6777 Abeysekera Kushala, 6214 Abiodun Phillip, 6857 Abouelnaga Ahmed, 5986 Abraham Nikhil, 6908 Absolam Sandra, 6294 Abwmdas Muhamad, 6042, 6565 Acar Sezer, 6687 Achara Chidinma, 6454 Adams Alexandra, 6146 Adamson Jonathan, 6312 Adarsh E, 6688 Addo-Quarshie Brenda, 6010 Adegoke Oluwakemi Tolu, 6560 Adeleye Oladunni, 6666 Adeyemi Ayopo, 6328 Adoghe Muwi, 6011 Agarwal Abhishek, 6264 Agrawal Shruti, 6625 Ahmad Fauzi Nur Azlyn, 6644 Ahmad Junaid, 6298 Ahmad Kajl, 6294 Ahmad Muhammad Hasnayn, 6561 Ahmadi Azita, 6336 Ahmed Abdalhakam, 6412 Ahmed Doaa, 6354 Ahmed Imad, 6427 Ahmed Leina, 6366 Ahmed Mansoor, 5988 Ahmed Mas, 6082, 6280, 6638 Ahmed Sarah, 6347 Ahmed Zoya, 6174 Aidoo Ella, 6669 Aina Blessing, 6792 Ajani Ayomikun, 6540 Ajayi Oluseyi, 5952, 6289, 6290, 6295 Ajayi-Obe Ekundayo, 6735 Ajmal Samira, 6313 Akcay Teoman, 6687 Akhtar Fauzia, 6204 Akintade Olayemi, 5977 Akter Subrina, 6879 Akyea Ralph Kwame, 6566 Al-Ani Shatha, 6724 Al-Dulaimi Hagir, 6136 Al-Muzaffar Iyad, 6013 Al-Nimri Omar M, 6410 Alabdullah Maryam, 6136 Aladangady Narendra, 6131, 6766 Aladi Sveta, 6807 Alberto Ianna, 5959 Albik Ahmad, 6535 Alderson Julie, 6896 Alexander Aiesha, 5964 Alghaeed Zubaida, 6042, 6565 Alghanim Salama, 6136 Alhadi Abdulhamid, 6042, 6565 Ali Afia, 6319 Ali May, 6724 Ali Muhammad, 6101 Ali Nusrat, 6820 Alicia.demirjian.gov.uk, 6127 Aljasmi Maryam, 6368 Alkamali Alya, 6368 Alkhdr Mahmoud Oklah, 5991 Alkotamy Marwa, 6158 Allen Deborah, 5973 Allen Zoe, 6045 Alloush Rasha, 6426 Allport Tom, 6256 Almoamen Nabeel, 6531 Alsatrawi Husain, 6531 Alsayigh Safaa, 5991 Alshafiei Latifa, 6368 Alshamari Thamir, 6706 Alsokhni Omar, 6583 Altaf Malik Kanwal, 6414 Aluvaala Jalemba, 6209 Amadi Anthony, 6061 Amadi-Livingstone Chibuchi, 6118 Amarakoon Givani, 6829, 6836, 6901 Ambegaonkar Gautam, 6696 Aminudin Nurul, 5976 Amirah Shakira, 6730 Amonoo-Kuofi Kwamena, 6802 Ampati Ioanna, 6771 Anand Binu, 6375 Anand Geetha, 6460 Ananthalingam Sweatha, 6080 Anbu Anbarasu Theodore, 6647 Anderson Anna-Karenia, 6798 Anderson Ruth, 6342 Andreski Lara, 6248 Andrew Odirichi, 6506 Andrews Clare, 6451, 6750 Ang Angelina, 6119 Ansell Karen, 6086 Anthony-Pillai Neil, 6628 Antony Tj, 6005 Anuruegbe Obinna, 6685 Appleby Jessica, 6068, 6429 Arend-Trujillo Monica, 6409 Argyropoulos Alexandros, 6099 Ariyaratnam Shaumya, 6164 Arlt Wiebke, 6902 Armitage Alice, 6515, 6684, 6705, 6848 Armitage Alice J, 6220 Armitage Suzanne, 6327 Armstrong Giles, 6611 Arora Rishi, 6313 Arora Sandeepa, 6475, 6710 Arsanious Meret, 6818 Arunath Visvalingam, 6379, 6513, 6517 Asamane Evans, 6512 Ashique Hana, 5964 Ashley Paul, 6266 Ashok Vidhu, 6397 Ashton Jill, 6642 Ashton Kirsty, 6315 Asimonye Ifunanya Scholastica, 6311 Asinobi Isaac Nwabueze, 6715 Assheton Philip, 6228 Aswani Nivedita, 6250, 6278 Aszkenasy Mark, 6590, 6591 Atar Muge, 6687 Athiraman Naveen, 6568 Attaalla Marina, 6615 Aunger Justin, 6512 Aurora Rosy, 6544 Austin Athira Mary, 6889 Authors GeNotes, 6242 Avenell Lucy, 6858 Awadh Asma, 6807 Awais Muhammad Azam, 6076, 6078 Ayak Blessing, 6548 Aye Mya, 6734 Aye Seinn Nwe, 6334 Aylett Sarah, 6870 Aylin Paul P, 6266 Ayya Mekhala, 6396 Ayyash Hani, 6448, 6846 Ayyash Hani F, 6410 Azam Huzaifa, 6105 Azevedo Marta Gomes de, 6606 Aziz Rafia, 6644 Azuka Nwaizu, 6645 Azuma Akiko, 6014 Babiker Mohamed OE, 6426, 6585, 6587 Babiker Mohammed OE, 6583 Bafna Vibha, 6239 Bagaber Omar, 6772 Baggott Lucy, 6246 Bahadursingh Prithiviraj, 6418 Bai Chadran, 6288 Baig Anya, 6408 Bailey Emma, 6307 Bailie Helen, 6429, 6888 Bains Manpreet, 6566 Baithun Christina, 6147 Bajaj Nivedita, 6449 Baker Alastair, 6348 Baker Camilla, 6616 Baker Catherine, 6109 Baker Elaine, 6194 Baker Elouise, 6790 Baker Kerry, 6120 Baker Robyn, 6303, 6315 Baki Yasmin, 6092, 6865, 6881 Bakshi Yogmaya, 6544 Bala Krishnan Ashanti Sham, 6572 Balakrishnan Vaisakh Sambasivan, 6636 Balasubramanian Ramnath, 6225 Ball Elizabeth, 6346 Ball Emily, 6637 Ball Emma, 6746, 6761 Ball Joanne, 6215 Ballard Mary-Rose, 6218 Ballot Daynia, 6289, 6290 Balmforth Sara, 6643 Bandaru Meghana, 6808 Banerjee Abir, 6724 Banerjee Kausik, 6724, 6771 Banerjee Tapomay, 6313, 6449 Bar Mohamed, 6559 Barber Alice, 6099 Bari Mohammad, 6313 Barnard Ed, 6058 Barnard Gemma, 6806 Barnard Lucy, 6098 Barnes Rebecca, 5944 Baron Madeleine, 6032 Barry Alexandra, 6186, 6187 Bartrum Nicola, 6107 Barwick Carol, 6339 Bas Serpil, 6687 Bashir Hana, 6270 Bashir Saadia, 6260 Basra Meirvaan, 6167 Bassett Paul, 6515 Basu Anna, 6707 Basu Aparajita, 6509 Bate Tristan, 6641 Batool Sidra Mehar, 6501 Batra Damini, 5969 Battersby Alexandra, 6800 Battersby Cheryl, 6441 Bawa Sara, 6581 Bayley Adam, 6281 Baynes Hannah, 6294 Beaney Thomas, 6698 Beardsall Kathryn, 6181 Beattie Thomas, 6447, 6452, 6453 Beattie Tom, 6186, 6187 Beatty Catherine, 6122, 6237 Beckett Robert, 6612 Bedford Helen, 6296 Bednarczuk Nadja, 5987, 6567 Beecham Aba, 6045 Beek Anna van, 6906 Beeston Gee-Na, 6377 Begum Fahmida, 6647 Bell Aaron, 6616 Bell Chris, 6248 Bell Joshua, 6228 Bellamy Johanna, 6228 Bello Oluwartimi, 6523 Bellsham-Revell Hannah, 6075 Belteki Gusztav, 6279, 6364 Bendapudi Perraju, 6287 Bendoni Hannah, 6498 Bensted Roland, 6264 Benzahia Abdurraouf, 6042, 6565 Benzaken Tami, 6750 Beqiri Sara, 6849 Berberoglu Merih, 6687 Berg Jonathan, 6374 Berg Richard, 6825 Bergero Georgina, 6721 Berki Alex, 6893 Besag Frank, 6449 Betts Georgina, 6668 Bhaduri Anjan, 6158 Bhagat Yesha, 6629 Bhagwat Bhanuja, 6239 Bhaiyat Raadhiyah, 5962 Bhaloo Nabeela, 6168 Bharadwaj Anurag, 6298 Bharat Rahul, 6218, 6516 Bhate Sanjay, 6744 Bhatia Sunanda, 6218 Bhattacharjee Anusha, 6329 Bhattacharyya Adrija, 6336 Bhawna Bhawna, 6005 Bibi Reena, 6122 Bickerton Georgia, 6735 Biggin-Lamming James, 6294 Bilgrami Syeda, 6530 Binham Lizzie, 6115 Birahinduka Dieudonne, 6071, 6615 Birch Simon, 6293 Bird Chris, 6174, 6312, 6350, 6773 Bisheswar Rhea, 6257, 6557 Bishop Michelle, 6242 Bisset Rachel, 6904 Biswas Taniya, 6725 Blair Mitch, 6430, 6515, 6705, 6750, 6848 Blair Mitchel, 6294 Blanquet Eloise, 6098 Blummers Sophie, 6248, 6790 Bogue Danielle, 6242 Bohatschet Marion, 6159 Bonfield Adam, 6101, 6764 Boroda Arlene, 6695 Borrill Heather, 6306 Bose Julian, 6131, 6766 Bostock Danielle, 6117 Bothamley Zoe, 6593, 6752 Bottle Alex, 6266, 6698 Boulton Anne, 6211 Boulton Grace, 6122 Bourke Thomas, 6610, 6649 Bouwers Deirdre, 6255 Bowyer Poppy, 6790 Boyes Megan, 6329 Braccio Serena, 6170 Bracken Abbey, 6471 Bracken Louise, 6320 Bradberry Sally, 6196 Brain Anna, 6354 Brandt-Varma Agnieszka, 6540 Brierley Joe, 6331, 6870, 6879 Brigham Ariel, 6215 Brinkley Becky, 6830 Broeze Carsten, 6286 Bromley-Brewes Kendal, 6871 Brookes Isobel, 6781 Brookes-Keene Richard, 6848 Brooks-Keene Richard, 6705 Broomfield Claire, 6858 Brown Katie, 6784 Brown Lisa, 6395 Brown Rosamund Casimir, 6608 Brown Sebastian, 6441 Brzyska Klara, 6066 Buckley Dannika, 6476 Buechner Hadassah, 6203, 6569 Bullock Kim, 6288 Bunch Amanda, 6035 Burfitt Elaine, 6711 Burford Anna, 6641 Burman Alisha, 6352, 6825 Burman Rhea, 6352 Burmeister Bruna, 6354 Burrati Giacomo, 6523 Burrill Amy, 6754 Burrows Mark, 6286 Burt Christine, 6760 Burton Katie, 6034 Butler Christopher C, 6040 Butler Mark, 6736 Butt Ayesha, 6353 Buxton Daniel, 6346 Cairns Lauren, 6802 Callaghan Bridget, 6458, 6895 Callow Ellis, 6834 Campbell Catherine, 6769 Cane Benada, 6294 Cannings-John Rebecca, 6040 Cardoso Pinto Alexandra M, 6336 Carpenter Bea, 6594 Carr Dominic, 6850 Carr Karen, 6437 Carr Siobhan, 6860 Carrasco Janette, 6461 Carrol Enitan D, 6040, 6471 Carroll Will, 6612 Carson-Stevens Andrew, 6637 Carstensen Maximilian, 6201 Carter Bernie, 6471 Carter Daniella, 5996, 6030 Carvalho Julene, 6523 Casey Annemarie, 6731 Cash Hannah, 6488 Cassim Sumaiya, 6898 Castelino Dipthi Nishal, 6434, 6811 Castillo Maria Del, 6320 Catli Gonul, 6687 Caygill Clare, 6339 Cecil Lizzie, 6833 Çelik İstemi Han, 6670 Cerra Kate, 6495 Chacko Shoba Mary, 5991 Chadha Komal, 6442 Chakkarapani Aravanan Anbu, 6102 Chalia Maria, 6161 Chan Jacquline, 6505 Chan Joe, 6324 Chan Ngai Man, 6464 Chana Shveta, 6118 Chandrasiri Suchitra, 6793 Chapman Lydia, 6324 Chapman Simon, 6294 Chathurika PTD, 6513 Chatoo Zaynab, 6831 Chattopadhyay Amitabha, 6420 Chaudhary Subhadra, 6747 Cheetham Tim, 6359 Chen Haoxin, 6714 Chencheri Nidheesh, 6136 Cheong Sabrina, 6202 Cherian Thomas, 6561 Chew Elaine, 6119 Chhabra Aakanksha, 6739 Chhapola Viswas, 6819 Chidambaranathan S, 5963 Chilukuri Lakshmi, 6592, 6618 Chimhuya Simbarashe, 6658 Ching Liew Christy Yie, 6271 Chinoy Amish, 6509 Chirwa Tobias, 6289, 6290 Chiume Msandeni, 6658 Chooi Stephen, 6013 Chowdhury Ashoka Dey, 6434 Chowdhury Olie, 6229 Chowdhury Riaz, 6680 Christian Peter, 6628 Chudleigh Jane, 6213 Chung Rosalind, 6045 Churchward Sarah, 6547 Cividini Sofia, 6803 Cizmecioglu Filiz, 6687 Clark Amy, 6040 Clark Andrew, 6876 Clark Simon, 6377, 6666 Clarke Jayne, 6754 Clesham Katherine, 6120 Coates Caroline, 6629 Coffey Frank, 6050 Cohen Danielle, 6596 Colbourne Tim, 6658 Cole Caroline, 6894 Cole Rachel, 6399 Colley Joanne, 6754 Collins Niamh, 6332 Collis Victoria, 6038 Connolly Paulina, 6796 Conway Tom, 6098 Cooksey Felicity, 6796 Coombes Lucy, 6213 Coombs Isha, 6451 Cooper Freya, 6461 Cooper Hannah, 6679 Cooper Lucy, 6902 Copeman Anna, 6696 Copeman Annabel, 6122, 6237 Cordingley Rebecca, 6575 Cordwell Jacinata, 6823 Corley Andrew, 6285 Corley Lisa, 6787 Cornaglia Francesca, 6129, 6192 Cornford Rosalind, 6430 Cosgrove Elin, 6395 Costa Cristine Sortica Da, 6211 Couch Helen, 6181 Coulson James, 6196 Cousins Katrina, 6696 Coutts Jonathan, 6144 Coverley Lydia, 6814 Cowling Isabel, 6837 Cox Olivia, 6886 Crabtree Nicola, 6396 Craig Kelly, 6872 Craig Stan, 6437 Crane Jo, 6293 Crawley Louise, 6521 Crawley Mollie, 6120 Creese Hanna, 6262 Critchl
Journal Article
Revisión Selectiva de los Tratamientos Psicológicos Empíricamente Apoyados Para Problemas de Salud Pediátricos
2025
DOI https://doi.org/10.70478/psicothema.2025.37.25Psicothema, 2025.Vol. 37 (nº 4). 16-28 PDFRevisión Selectiva de los Tratamientos Psicológicos Empíricamente Apoyados Para Problemas de Salud PediátricosSelective Review of Empirically Supported Psychological Treatments for Pediatric Health ProblemsIsabel Cuéllar-Flores1, Izaskun Basterra2, Silvia Cámara3, Borja Esteso-Orduña4, Deborah Fuentes-Casany5, Laura González-Riesco6, Amaia Izquierdo7, María del Pilar Mundo-Cid8, Estela Salcedo9, Raquel Seijas-Gómez10 & María Serrano-Villar111 Hospital Clínico San Carlos (Spain) 2 Hospital Universitario de Navarra (Spain) 3 Hospital General Universitario Gregorio Marañón (Spain) 4 Hospital Infantil Universitario Niño Jesús (Spain) 5 Institut d’Assistència Sanitària (Spain) 6 Hospital Sant Joan de Déu (Spain) 7 Hospital Río Hortega (Spain) 8 Hospital Universitari Germans Trias i Pujol (Spain) 9 Hospital Universitario Clínico San Cecilio (Spain) 10 Hospital Teresa Herrera (Spain) 11 Hospital de Getafe (Spain)ABSTRACT Background: Significant progress has been made in providing empirical support for psychological interventions designed to improve the health and behavior of children and young people with pediatric health problems. The objective of this study was to carry out a review of empirically supported psychological treatments for various health problems in the child and adolescent population. Method: Psychological treatments with empirical support for 12 pediatric health problems were reviewed based on the levels of evidence and how much they are recommended in the Spanish national health system Clinical Practice Guides. Results: Psychological treatments to address various health problems in the child and adolescent population have empirical support. The level of evidence ranges from low to high depending on the health problem. The review shows uneven deployment in different health problems. Conclusions: This review will help psychology professionals and healthcare managers to make decisions regarding psychological treatments for addressing pediatric health problems.RESUMEN Antecedentes: Se han logrado avances significativos en proporcionar apoyo empírico para intervenciones psicológicas diseñadas para mejorar la salud de niños/as y jóvenes que presentan problemas de salud pediátricos. El objetivo de este trabajo ha sido realizar una revisión de los tratamientos psicológicos empíricamente apoyados para el abordaje de diferentes problemas de salud en población infanto-juvenil. Método: Se han revisado los tratamientos psicológicos con apoyo empírico para 12 problemas de salud pediátricos en función de los niveles de evidencia y grados de recomendación de las Guías de Práctica Clínica del Sistema Nacional de Salud de España. Resultados: Los tratamientos psicológicos para el abordaje de varios problemas de salud en población infanto-juvenil cuentan con apoyo empírico. El nivel de evidencia oscila de bajo a alto en función del problema de salud. La revisión muestra un desarrollo desigual en los diferentes problemas de salud. Conclusiones: Esta revisión ayudará a los profesionales de la psicología y gestores en la toma de decisiones con respecto a los tratamientos psicológicos para el abordaje de los problemas de salud pediátricos.PDF English English
Journal Article
P-031 The orbital grading system performance in assessing angiographic outcomes for moyamoya vasculopathy in children: an inter-rater reliability analysis
2025
BackgroundThe Matsushima grading system is widely used to evaluate revascularization outcomes after Encephalo-Duro-Arterio-Synangiosis (EDAS) for Moyamoya vasculopathy by describing collateral ingrowth from the superficial temporal artery (STA) to the MCA territory. However, its subjective measurement and the potential ingrowth of collaterals to the ACA territory, as well as those from the MMA, are all limitations of the scale. The orbital grading system (OGS) is a new, objective scale initially developed to measure these outcomes in adults. We aim to evaluate this scale in the pediatric population.MethodsRetrospective analysis of patients with Moyamoya disease and syndrome treated with EDAS at a single major pediatric center from 2006–2023. An inter-rater reliability analysis was performed using 5 raters with different levels of training that provided scores for 30 blinded cases, and then a Fleiss’ kappa coefficient (k) was calculated as a measure of agreement beyond chance.Results101 patients with a mean age of 9 years old, mostly females (56.4%) and mostly comprised of Moyamoya disease (71.3%), were included. A total of 158 cerebral hemispheres were treated with EDAS (57 patients had bilateral surgeries). Most patients were categorized as Suzuki grade III or above (82.9%). At a median of 12.4 months, collateral growth graded using the Matsushima scale was A in 57.6%, B in 24%, C in 14.6%, and borderline grades in 3.8%. The OGS reported scores were grade 0 (7.0%), grade 1 (8.9%), grade 2 (43.7%), and grade 3 (40.5%). inter-rater agreement evaluation found that the OGS had a superior Kappa than the Matsushima Grading (0.51 vs. 0.15; p < 0.001).ConclusionThe OGS had higher inter-rater agreement rates compared to the Matsushima scale for identifying postoperative collateral ingrowth in children after EDAS. This scale offers a more objective way to evaluate radiological outcomes after EDAS for the pediatric population.DisclosuresA. Enriquez-Marulanda: None. M. Ruppert-Gomez: None. P. Lazatin: None. S. Staffa: None. S. Rangwala: None. D. Orbach: None. A. See: None. E. Smith: None. C. Ogilvy: None.
Journal Article
Liraglutide for Children 6 to <12 Years of Age with Obesity — A Randomized Trial
by
Barrientos-Pérez, Margarita
,
Jalaludin, Muhammad Yazid
,
Sahu, Kushal
in
Adolescent Medicine
,
Adverse events
,
Anti-Obesity Agents - administration & dosage
2025
Obesity often develops in childhood. In children (6 to <12 years of age) with obesity, treatment with liraglutide for 56 weeks plus lifestyle interventions induced a greater mean change in BMI than lifestyle interventions alone.
Journal Article
82 Assessing the usage of and barriers to using language interpretation services in a Canadian tertiary paediatric center
2024
Abstract
Background
Canada has one of the highest immigration rates worldwide per population with roughly 447,180 permanent residents welcomed in 2022. Limited-English proficiency (LEP) populations face barriers in accessing health care with implications including delayed presentation, misdiagnosis, poor compliance, decreased patient comprehension, and adverse events. Adequate health care provider training in using language interpretation services (LIS) is crucial to improve the quality of patient care and reduce miscommunication. There is a lack of research in this area in Canadian paediatric populations.
Objectives
To identify attitudes towards and usage of LIS among health care providers in a tertiary paediatric center, as well as levels of LIS training received.
Design/Methods
An online survey was developed and distributed to paediatric health care professionals including physicians, residents/fellows, nursing staff, and allied health providers. Survey completion was anonymous and voluntary; data collection occurred from October 2022–February 2023. Descriptive statistics were used for quantitative data and thematic analysis for free text responses.
Results
345 respondents accessed the survey and 281(81%) completed it. Multiple paediatric professions were surveyed with most responses stemming from nursing staff, residents and physicians, and other allied health. 52.8% of respondents encountered a paediatric patient and/or their caregivers with a language barrier in English once per week or more. LIS were used by 53.1% of respondents once per week or more. 51.5% of respondents reported no previous training in using LIS in patient care, and 3.6% reported previous formal training. 57.9% of respondents felt LIS training was very beneficial to their role and 53.7% reported this increased their LIS usage. Overall, in-person interpreters were perceived as the most effective LIS modality followed by video and telephone interpreters. Barriers to using LIS included: inability to access desired modality and/or language; insufficient time; and lack of comfort in effective usage.
Conclusion
It was evident respondents felt LIS was integral to patient care, especially with an increasingly diverse population. Multiple barriers to using LIS were identified with likely applicability to other Canadian paediatric facilities. Suggested improvements were themed around improving LIS accessibility, improving LIS education and awareness, and increasing access to rare languages. This study has important implications for LIS and paediatric care, including informing educational and advocacy initiatives, policy change, and LIS resource allocation.
Journal Article
MON-474 Adrenal Control in Pediatric 11β-Hydroxylase Deficient CAH Patients Using Different Hydrocortisone Formulations: A Two-Patient Case Study
2025
Abstract
Disclosure: P. Seo: None. M.M. Rutter: None. M. Yau: None.
The classical form of 11β-hydroxylase deficiency (11-β OHD) CAH is differentiated by severe hypertension due to the mineralocorticoid effect of deoxycorticosterone and hyperandrogenism. For children, the Endocrine Society recommends daily hydrocortisone (HC) at 10-15 mg/m^2/day in divided doses. As children grow, frequent biological and laboratory monitoring is needed for small-dose titrations of HC to optimize adrenal control. Oral HC tablets are not explicitly designed for children, necessitating splitting tablets or having pediatric-specific formulations compounded. This process makes it challenging to deliver consistent doses, particularly for amounts less than 5 mg. In late 2020, the US FDA approved a pediatric-specific HC formulation in oral granules, with doses as low as 0.5 mg. Objective To assess adrenal control in CAH on various formulations of HC, we retrospectively reviewed the medical records of two pediatric patients with 11-β OHD who had received different formulations of oral HC (solution, tablets, and oral granules) for up to 4.5 years. Methods We collected demographic, anthropometric, and pubertal data, laboratory results, and medication formulation and dosing over multiple time points during pediatric endocrine follow-up. Laboratory results included ACTH, androstenedione, plasma renin activity (PRA), deoxycorticosterone, and 11-deoxycortisol concentrations. Choice of HC oral formulations was based on family preference and provider recommendation. Results Patient 1 (27-53 mo) was initially prescribed HC solution at doses of 8.5 -14.3 mg/m^2/day and transitioned to granules at 41 mo at 11.7-14.6 mg/m^2/day. Adrenal control on HC solution and granules was similar on solution and granules with androstenedione of 31-56 ng/dl, PRA of 0.17-2.7 . ACTH peaked at 54.8 pg/mL at 38 mo But normalized over time. Androstenedione stayed within the normal range, while 11-deoxycortisol peaked at 232 ng/dL. PRA remained below normal, except at 41 mo. Patient 2 (5-39 mo) transitioned from HC solution at 18 mg/m^2/day to tablets at 16-19 mg/m^2/day at 9 mo and then to granules at 14 m.o. While on granules, HC doses decreased from 18.2 to 7.25 mg/m^2/day due to poor compliance. Until 30 mo, ACTH and androstenedione remained low, respectively at<6 pg/mL) and <10 ng/dL). PRA was consistently below the normal range, and 11-deoxycortisol spiked at 30 mo (2029 ng/dL) and androstenedione rose to 115 ng/dl at 39 mo. Changes in formulations were well tolerated. Conclusion The results indicate that adrenal control was influenced by factors beyond the formulation of HC. Variations in adrenal control were observed based on the dosage of HC relative to body surface area, with the total daily dose emerging as a primary determinant of adrenal regulation. Adherence with medication dosing and stressful blood draws may have contributed to poor adrenal control and should be explored further.
Presentation: Monday, July 14, 2025
Journal Article