Real-World Estimates of Adrenal Insufficiency-Related Adverse Events in Children With Congenital Adrenal Hyperplasia

被引：27

作者：

Ali, Salma R. ^{[1
,2
,3
]}

Bryce, Jillian ^{[2
,3
]}

Haghpanahan, Houra ^{[4
]}

Lewsey, James D. ^{[4
]}

Tan, Li En ^{[1
]}

Atapattu, Navoda ^{[5
]}

Birkebaek, Niels H. ^{[6
]}

Blankenstein, Oliver ^{[7
]}

Neumann, Uta ^{[7
]}

Balsamo, Antonio ^{[8
]}

Ortolano, Rita ^{[8
]}

Bonfig, Walter ^{[9
,10
]}

Claahsen-van der Grinten, Hedi L. ^{[11
]}

Cools, Martine ^{[12
]}

Costa, Eduardo Correa ^{[13
]}

Darendeliler, Feyza ^{[14
]}

Poyrazoglu, Sukran ^{[14
]}

Elsedfy, Heba ^{[15
]}

Finken, Martijn J. J. ^{[16
]}

Fluck, Christa E. ^{[17
,18
]}

Gevers, Evelien ^{[19
]}

Korbonits, Marta ^{[19
]}

Guaragna-Filho, Guilherme ^{[20
]}

Guran, Tulay ^{[21
]}

Guven, Ayla ^{[22
]}

Hannema, Sabine E. ^{[23
,24
]}

Higham, Claire ^{[25
]}

Hughes, Ieuan A. ^{[26
]}

Tadokoro-Cuccaro, Rieko ^{[26
]}

Thankamony, Ajay ^{[26
]}

Iotova, Violeta ^{[27
]}

Krone, Nils P. ^{[28
]}

Krone, Ruth ^{[29
]}

Lichiardopol, Corina ^{[30
]}

Luczay, Andrea ^{[31
]}

Mendonca, Berenice B. ^{[32
]}

Bachega, Tania A. S. S. ^{[32
]}

Miranda, Mirela C. ^{[32
]}

Milenkovic, Tatjana ^{[33
]}

Mohnike, Klaus ^{[34
]}

Nordenstrom, Anna ^{[35
]}

Einaudi, Silvia ^{[36
]}

van der Kamp, Hetty ^{[37
]}

Vieites, Ana ^{[38
]}

de Vries, Liat ^{[39
,40
]}

Ross, Richard J. M. ^{[28
]}

Ahmed, S. Faisal ^{[1
,2
,3
]}

机构：

[1] Univ Glasgow, Sch Med Dent & Nursing, Dev Endocrinol Res Grp, Glasgow G51 4TF, Lanark, Scotland

[2] Royal Hosp Children, Off Rare Condit, Glasgow G51 4TF, Lanark, Scotland

[3] Queen Elizabeth Univ Hosp, Glasgow G51 4TF, Lanark, Scotland

[4] Univ Glasgow, Inst Hlth & Wellbeing, Hlth Econ & Hlth Technol Assessment, Glasgow G12 8RZ, Lanark, Scotland

[5] Lady Ridgeway Hosp, Colombo 00800, Sri Lanka

[6] Aarhus Univ Hosp, Dept Paediat, DK-8200 Aarhus, Denmark

[7] Charite Univ Med Berlin, Ctr Chron Sick Children, Inst Expt Paediat Endocrinol, D-10117 Berlin, Germany

[8] S Orsola Malpighi Univ Hosp, Ctr Rare Endocrine Condit Endo ERN, Dept Med & Surg Sci, Pediat Unit, I-40138 Bologna, Italy

[9] Tech Univ Munich, Dept Paediat, D-80804 Munich, Germany

[10] Klinikum Wels Grieskirchen, Dept Paediat, A-4600 Wels, Austria

[11] Radboud Univ Nijmegen, Amalia Childrens Hosp, Dept Paediat Endocrinol, Med Ctr, NL-6525 GA Nijmegen, Netherlands

[12] Univ Ghent, Univ Hosp Ghent, B-9000 Ghent, Belgium

[13] Univ Fed Rio Grande do Sul, Pediat Surg Serv, Hosp Clin Porto Alegre, BR-90035903 Porto Alegre, RS, Brazil

[14] Istanbul Univ, Istanbul Fac Med, Dept Paediat, Paediat Endocrinol Unit, TR-34093 Istanbul, Turkey

[15] Ain Shams Univ, Dept Pediat, Cairo 11566, Egypt

[16] Vrije Univ Amsterdam, Emma Childrens Hosp, Pediat Endocrinol, Amsterdam UMC, NL-1105 AZ Amsterdam, Netherlands

[17] Univ Bern, Inselspital, Bern Univ Hosp, Pediat Endocrinol Diabetol & Metab,Dept Pediat, CH-3010 Bern, Switzerland

[18] Univ Bern, Inselspital, Bern Univ Hosp, Dept BioMed Res, CH-3010 Bern, Switzerland

[19] Queen Mary Univ London, Barts & London Sch Med, William Harvey Res Inst, Dept Endocrinol, London EC1M 6BQ, England

[20] Univ Fed Rio Grande do Sul UFRGS, Sch Med, Dept Pediat, BR-90035903 Porto Alegre, RS, Brazil

[21] Marmara Univ, Dept Pediat Endocrinol & Diabet, TR-34899 Istanbul, Turkey

[22] Hlth Sci Univ, Zeynep Kamil Women & Children Hosp, Med Fac, Pediat Endocrinol Clin, TR-34668 Istanbul, Turkey

[23] Sophia Childrens Univ Hosp, Erasmus Med Ctr, Dept Paediat Endocrinol, NL-3015 CN Rotterdam, Netherlands

[24] Leiden Univ, Dept Paediat, Med Ctr, NL-2333 ZA Leiden, Netherlands

[25] Univ Manchester, Christie Hosp NHS Fdn Trust, Manchester Acad Hlth Sci Ctr, Dept Endocrinol, Manchester M20 4BX, Lancs, England

[26] Univ Cambridge, Dept Paediat, Cambridge CB2 2QQ, England

[27] Med Univ Varna, Dept Paediat, UMHAT Sv Marina, Varna 9002, Bulgaria

[28] Univ Sheffield, Dept Oncol & Metab, Sheffield S10 2RX, S Yorkshire, England

[29] Birmingham Womens & Childrens Hosp, Dept Endocrinol & Diabet, Birmingham B4 6NH, W Midlands, England

[30] Univ Med & Pharm Craiova, Univ Emergency Hosp, Dept Endocrinol, Craiova 200349, Romania

[31] Semmelweis Univ, Dept Paediat, H-1085 Budapest, Hungary

[32] Univ Sao Paulo, Hosp Clin, Fac Med,Disciplina Endocrinol, Lab Hormonios & Genet Mol LIM42,Unidade Endocrino, Sao Paulo, SP, Brazil

[33] Mother & Child Hlth Care Inst Serbia Dr Vukan Cup, Dept Endocrinol, Belgrade, Serbia

[34] Univ Magdeburg, D-39106 Magdeburg, Germany

[35] Karolinska Univ Hosp, SE-17177 Stockholm, Sweden

[36] Univ Turin, Pediat Endocrinol Regina Margherita Childrens Hos, Citta Salute & Sci, I-10126 Turin, Italy

[37] Wilhelmina Childrens Hosp, Div Pediat Endocrinol, NL-3584 EA Utrecht, Netherlands

[38] Hosp Ninos Dr Ricardo Gutierrez, Ctr Invest Endocrinol, Div Endocrinol, RA-1425 Buenos Aires, DF, Argentina

[39] Schneider Childrens Med Ctr Israel, Jesse & Sara Lea Shafer Inst Endocrinol & Diabet, IL-4920235 Petah Tiqwa, Israel

[40] Tel Aviv Univ, Sackler Fac Med, IL-6997801 Tel Aviv, Israel

来源：

JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM | 2021年 / 106卷 / 01期

基金：

英国医学研究理事会;

关键词：

21-hydroxylase deficiency; adrenal insufficiency; adrenal crisis; congenital adrenal hyperplasia; registry; CRISIS; MANAGEMENT; ADOLESCENTS; EXPERIENCE;

D O I：

10.1210/clinem/dgaa694

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Background: Although congenital adrenal hyperplasia (CAH) is known to be associated with adrenal crises (AC), its association with patient- or clinician-reported sick day episodes (SDE) is less clear. Methods: Data on children with classic 21-hydroxylase deficiency CAH from 34 centers in 18 countries, of which 7 were Low or Middle Income Countries (LMIC) and 11 were High Income (HIC), were collected from the International CAH Registry and analyzed to examine the clinical factors associated with SDE and AC. Results: A total of 518 children-with a median of 11 children (range 1, 53) per center-had 5388 visits evaluated over a total of 2300 patient-years. The median number of AC and SDE per patient-year per center was 0 (0, 3) and 0.4 (0.0, 13.3), respectively. Of the 1544 SDE, an AC was reported in 62 (4%), with no fatalities. Infectious illness was the most frequent precipitating event, reported in 1105 (72%) and 29 (47%) of SDE and AC, respectively. On comparing cases from LMIC and HIC, the median SDE per patient-year was 0.75 (0, 13.3) vs 0.11 (0, 12.0) (P < 0.001), respectively, and the median AC per patient-year was 0 (0, 2.2) vs 0 (0, 3.0) (P = 0.43), respectively. Conclusions: The real-world data that are collected within the I-CAH Registry show wide variability in the reported occurrence of adrenal insufficiency-related adverse events. As these data become increasingly used as a clinical benchmark in CAH care, there is a need for further research to improve and standardize the definition of SDE.

引用

页码：E192 / E203

页数：12

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