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Article ; Online: Peripheral Arrangement of Steatosis Microvacuoles in Wilson's Disease.

Laurent-Bellue, Astrid / Sobesky, Rodolphe / Guettier, Catherine

Clinical gastroenterology and hepatology : the official clinical practice journal of the American Gastroenterological Association

2021 Volume 20, Issue 4, Page(s) A17

MeSH term(s)	Fatty Liver/diagnosis ; Hepatolenticular Degeneration/diagnosis ; Humans
Language	English
Publishing date	2021-08-05
Publishing country	United States
Document type	Journal Article
ZDB-ID	2119789-1
ISSN	1542-7714 ; 1542-3565
ISSN (online)	1542-7714
ISSN	1542-3565
DOI	10.1016/j.cgh.2021.08.005
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Article ; Online: Efficiency and safety of total plasma exchange in critically ill cirrhotic patients with acute on chronic liver failure: A pilot study.

Kounis, Ilias / Sacleux, Sophie Caroline / Ordan, Marie Amelie / André, Stéphane / Boudon, Marc / Coilly, Audrey / Sobesky, Rodolphe / De Martin, Eleonora / Samuel, Didier / Ichaï, Philippe / Saliba, Faouzi

Clinics and research in hepatology and gastroenterology

2023 Volume 47, Issue 8, Page(s) 102206

Abstract: Background and aims: Treatment of patients with acute on chronic liver failure (ACLF) admitted to the ICU is very limited. The aim of this pilot study was to evaluate the efficiency on liver function and safety of therapeutic plasma exchange (TPE) in ... ...

Abstract	Background and aims: Treatment of patients with acute on chronic liver failure (ACLF) admitted to the ICU is very limited. The aim of this pilot study was to evaluate the efficiency on liver function and safety of therapeutic plasma exchange (TPE) in critically ill cirrhotic patients admitted with ACLF in a liver ICU. Methods: This is a prospective cohort of patients with ACLF grade > 2 treated by TPE admitted to the ICU that was matched to a control group. TPE was performed using a plasma filter (TPE2000, BAXTER®) on a CRRT machine (Prismaflex®, Baxter®). Ratio and type of fluid replacement were 50 % with 5 % albumin solution followed by 50 % with fresh frozen plasma. Results: Seven patients with a mean age of 50.6 ± 7.8 years (all males) and 14 controls matched to age, sex, etiology and cause of decompensation were recruited. At ICU admission, mean MELD score was 39.1 ± 2.7, mean SOFA score was 11.6 ± 5.2 and mean CLIF SOFA score was 12.9 ± 2.6. The grade of ACLF was 3 for 3 patients (42.9 %) and 2 for 4 patients (57.1 %). The TPE group had significantly higher levels of bilirubin (392.3 ± 117.1μmol/l vs. 219 ± 185μmol/l , p = 0.04), and INR values (5.7 ± 3.4 vs. 3.5 ± 0.9, p < 0.005) compared to the control group. Patient survival was respectively 28.6 % and 14.3 % at 30 and 90 days in the TPE group and 35.7 % and 7.14 % in the control group respectively (HR: 1 (95 % CI 0.19- 5.2; p = 1). One patient in the TPE group had a liver transplantation 13 days after admission to ICU and is still alive and none in the control group. Two (28.6 %) patients died from complications related to the double lumen catheter used for TPE. Conclusion: This pilot study of TPE in patients with ACLF grade 2 and 3 showed a marked but transient improvement in liver function tests. TPE worth to be evaluated in large trials in ACLF patients, with a liver transplant project, and less organ failure.
Language	English
Publishing date	2023-09-14
Publishing country	France
Document type	Journal Article
ZDB-ID	2594333-9
ISSN	2210-741X ; 2210-7401
ISSN (online)	2210-741X
ISSN	2210-7401
DOI	10.1016/j.clinre.2023.102206
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Article ; Online: Nodular Regenerative Hyperplasia Is Not a Rare Condition After Liver Transplantation: Incidence, Predictive Factors, and Impact on Survival.

Kounis, Ilias / Sebagh, Mylène / Evain, Manon / Cailliez, Valérie / Roche, Bruno / De Martin, Eleonora / Sobesky, Rodolphe / Guettier, Catherine / Allard, Marc Antoine / Golse, Nicolas / Azoulay, Daniel / Vibert, Eric / Duclos Vallee, Jean Charles / Feray, Cyrille / Samuel, Didier / Coilly, Audrey

Transplantation

2023 Volume 107, Issue 2, Page(s) 410–419

Abstract: Background: The objectives of this study were to evaluate incidence and to identify the risk factors of occurrence and the predictive factors of symptomatic forms of nodular regenerative hyperplasia (NRH) after liver transplantation (LT).: Methods: ... ...

Abstract	Background: The objectives of this study were to evaluate incidence and to identify the risk factors of occurrence and the predictive factors of symptomatic forms of nodular regenerative hyperplasia (NRH) after liver transplantation (LT). Methods: To identify risk factors of NRH following LT, we included 1648 patients transplanted from 2004 to 2018 and compared the patients developing NRH after LT to those who did not. To identify predictive factors of symptomatic NRH, we selected 115 biopsies displaying NRH and compared symptomatic to asymptomatic forms. Symptomatic NRH was defined as the presence of ascites, esophageal varices, hepatic encephalopathy, portal thrombosis, retransplantation, or death related to NRH. Results: The incidence of NRH following LT was 5.1%. In multivariate analysis, the independent factor of developing NRH after LT was the donor's age (odds ratio [OR] = 1.02; confidence interval, 1.01-1.03; P = 0.02). Symptomatic forms occurred in 29 (25.2%) patients: 19 (16.5%) patients presented with ascites, 13 (11.3%) with esophageal varices, 4 (3.5%) with hepatic encephalopathy, and 8 (7%) with portal thrombosis. The median period before the onset of symptoms was 8.4 (1.5-11.3) y after LT. The spleen size at diagnosis/before LT ratio (OR = 12.5; 114.17-1.37; P = 0.0252) and thrombectomy during transplantation (OR = 11.17; 1.48-84.11; P = 0.0192) were associated with symptomatic NRH in multivariate analysis. Conclusions: NRH following LT is frequent (5.1%) and leads to symptomatic portal hypertension in 25.2% of patients. Using older grafts increases the risk of developing NRH after LT. Clinicians should screen for signs of portal hypertension, particularly in measuring spleen size.
MeSH term(s)	Humans ; Liver Transplantation/adverse effects ; Liver/pathology ; Hyperplasia/complications ; Hyperplasia/pathology ; Esophageal and Gastric Varices/complications ; Esophageal and Gastric Varices/pathology ; Ascites/epidemiology ; Ascites/etiology ; Hepatic Encephalopathy/complications ; Hepatic Encephalopathy/pathology ; Incidence ; Hypertension, Portal/diagnosis ; Hypertension, Portal/epidemiology ; Hypertension, Portal/etiology ; Thrombosis/pathology
Language	English
Publishing date	2023-01-26
Publishing country	United States
Document type	Journal Article
ZDB-ID	208424-7
ISSN	1534-6080 ; 0041-1337
ISSN (online)	1534-6080
ISSN	0041-1337
DOI	10.1097/TP.0000000000004303
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Zs.A 488: Show issues			Location: Je nach Verfügbarkeit (siehe Angabe bei Bestand) bis Jg. 1994: Bestellungen von Artikeln über das Online-Bestellformular Jg. 1995 - 2021: Lesesall (1.OG) ab Jg. 2022: Lesesaal (EG)
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Article: Impact of body mass index on hepatocellular carcinoma recurrence after liver transplantation through long-term follow-up.

El-Domiaty, Nada / Saliba, Faouzi / Karam, Vincent / Sobesky, Rodolphe / Ibrahim, Wafaa / Vibert, Eric / Pittau, Gabriella / Amer, Khaled / Saeed, Maysaa A / Shawky, Jihan A / Cherqui, Daniel / Adam, René / Samuel, Didier

Hepatobiliary surgery and nutrition

2021 Volume 10, Issue 5, Page(s) 598–609

Abstract: Background: Obesity is associated with increased oncological risk and outcomes but the evidence surrounding the effect of body mass index (BMI) on increased risk of hepatocellular carcinoma (HCC) recurrence after liver transplantation (LT) is still ... ...

Abstract	Background: Obesity is associated with increased oncological risk and outcomes but the evidence surrounding the effect of body mass index (BMI) on increased risk of hepatocellular carcinoma (HCC) recurrence after liver transplantation (LT) is still questionable. The purpose of this retrospective study of a large cohort of adult patients transplanted for HCC was to investigate the effect of BMI on the incidence of HCC recurrence and outcome. Methods: Data from 427 adult recipients transplanted for HCC between 2000 and 2017 were collected. Patients were classified at time of LT according to the World Health Organization BMI classification into 3 groups; group 1: BMI <25 (n=166), group 2: BMI 25-29.9 (n=150) and group 3: BMI ≥30 (n=111). Results: There were no significant changes of mean BMI overtime 26.8±5.0 kg/m2 at time of LT and 28.8±23.1 at 5 years. The recurrence rates of HCC after LT in the three groups were 19%, 16% and 17% respectively. The 5, 10 and 15-year recurrence free survival (RFS) rates were respectively 68.6%, 47.3% and 40.8% in group 1, 73.3%, 66.2% and 49.5% in group 2 and 68.8%, 57.5% and 47.7% in group 3 (log rank P=0.47). Conclusions: Recipient BMI at time of transplant and during follow-up didn't impact the incidence of HCC recurrence nor long-term patient survival, irrespective to the status of the patients and their tumor characteristic at time of LT. The present study clearly confirms that obesity should not be considered, when selecting patients with HCC to LT, as a predictive factor of recurrence.
Language	English
Publishing date	2021-07-12
Publishing country	China (Republic : 1949- )
Document type	Journal Article
ZDB-ID	2812398-0
ISSN	2304-389X ; 2304-3881
ISSN (online)	2304-389X
ISSN	2304-3881
DOI	10.21037/hbsn.2020.04.01
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Article ; Online: Liver transplant selection criteria and outcomes in critically ill patients with ACLF.

JHEP reports : innovation in hepatology

2023 Volume 6, Issue 1, Page(s) 100929

Abstract: Background & aims: Retrospective studies have reported good results with liver transplantation (LTx) for acute-on-chronic liver failure (ACLF) in selected patients. The aim of this study was to evaluate the selection process for LTx in patients with ... ...

Abstract	Background & aims: Retrospective studies have reported good results with liver transplantation (LTx) for acute-on-chronic liver failure (ACLF) in selected patients. The aim of this study was to evaluate the selection process for LTx in patients with ACLF admitted to the intensive care unit (ICU) and to assess outcomes. Methods: This prospective, non-interventional, single high-volume center study collected data on patients with ACLF admitted to the ICU between 2017-2020. Results: Among 200 patients (mean age: 55.0 ± 11.2 years and 74% male), 96 patients (48%) were considered potential candidates for LTx. Unfavourable addictology criteria (n = 76) was the main reason for LTx ineligibility. Overall, 69 patients were listed for LTx (34.5%) and 50 were transplanted (25% of the whole population). The 1-year survival in the LTx group was significantly higher than in the non-transplanted group (94% Conclusion: This prospective analysis of outcomes of patients with ACLF admitted to the ICU highlights the drastic nature of selection in this setting. Unfavourable addictology criteria, mechanical ventilation and increasing number of organ failures since admission were predictive of absence of LTx, futility and death. Impact and implications: Liver transplantation (LT) is the best therapeutic option in selected cirrhotic patients admitted to the ICU with acute on chronic liver failure. However, the selection criteria are poorly described and based on retrospective studies. This is the first prospective study that aimed to describe the selection process for LT in a transplant center. Patients with ACLF should be admitted to the ICU and evaluated within a short period of time for LT. In the context of organ shortage, eligibility for LT and either absence of LT, futility of care or death are better clarified in our study. These are mainly determined by prolonged respiratory failure and worsening of organ failures since ICU admission. Considering worldwide variations in the etiology and definition of ACLF, transplant availability and a narrow therapeutic window for transplant further prospective studies are awaited.
Language	English
Publishing date	2023-10-20
Publishing country	Netherlands
Document type	Journal Article
ISSN	2589-5559
ISSN (online)	2589-5559
DOI	10.1016/j.jhepr.2023.100929
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Article ; Online: Non-invasive diagnosis and follow-up of rare genetic liver diseases.

Sobesky, Rodolphe / Guillaud, Olivier / Bouzbib, Charlotte / Sogni, Philippe / Poujois, Aurélia / Woimant, France / Duclos-Vallée, Jean Charles / Bourlière, Marc / de Lédinghen, Victor / Ganne-Carrié, Nathalie / Bureau, Christophe

Clinics and research in hepatology and gastroenterology

2021 Volume 46, Issue 1, Page(s) 101768

Abstract: Rare genetic liver diseases can result in multi-systemic damage, which may compromise the patient's prognosis. Wilson's disease and alpha-1 antitrypsin deficiency must be investigated in any patient with unexplained liver disease. Cystic fibrosis ... ...

Abstract	Rare genetic liver diseases can result in multi-systemic damage, which may compromise the patient's prognosis. Wilson's disease and alpha-1 antitrypsin deficiency must be investigated in any patient with unexplained liver disease. Cystic fibrosis screening of new-borns is now implemented in most high-prevalence countries. The diagnosis of these diseases can be strongly suggested with specific non-invasive tests. Molecular analysis gene for these diseases is long and tedious but is recommended to confirm the diagnosis and help for the family screening. Liver biopsy is not systematic and is discussed when it helps diagnosis. Currently, for these three diseases, non-invasive fibrosis markers could identify patients with risk of cirrhosis and complications. Rare genetic liver diseases can result in multi-systemic damage, which may compromise the patient's prognosis. Wilson's disease, must be investigated in any patient with unexplained liver disease and/or unexplained neurological or neuropsychiatric disorders. The diagnosis is based on a combination of clinical, biological features, including copper balance. The exchangeable copper/total copper ratio is a new sensible and specific biological marker, useful for the diagnosis of the disease. Timely diagnosis and treatment will prevent serious complications from the disease. Neurological evaluation and familial screening are essential in patients with Wilson's disease.
MeSH term(s)	Biomarkers ; Copper ; Follow-Up Studies ; Hepatolenticular Degeneration/diagnosis ; Hepatolenticular Degeneration/genetics ; Humans ; Liver ; Rare Diseases ; alpha 1-Antitrypsin Deficiency/complications ; alpha 1-Antitrypsin Deficiency/diagnosis ; alpha 1-Antitrypsin Deficiency/genetics
Chemical Substances	Biomarkers ; Copper (789U1901C5)
Language	English
Publishing date	2021-07-29
Publishing country	France
Document type	Journal Article ; Practice Guideline
ZDB-ID	2594333-9
ISSN	2210-741X ; 2210-7401
ISSN (online)	2210-741X
ISSN	2210-7401
DOI	10.1016/j.clinre.2021.101768
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Article ; Online: Diagnosis and Outcomes of Late-Onset Wilson's Disease: A National Registry-Based Study.

Nilles, Christelle / Obadia, Mickael Alexandre / Sobesky, Rodolphe / Dumortier, Jérôme / Guillaud, Olivier / Laurencin, Chloé / Moreau, Caroline / Vanlemmens, Claire / Ory-Magne, Fabienne / de Ledinghen, Victor / Bardou-Jacquet, Edouard / Fluchère, Frederique / Collet, Corinne / Oussedik-Djebrani, Nouzha / Woimant, France / Poujois, Aurélia

Movement disorders : official journal of the Movement Disorder Society

2022 Volume 38, Issue 2, Page(s) 321–332

Abstract: Background: Wilson's disease (WD) is usually diagnosed in children and young adults; limited data exist on late-onset forms.: Objective: The aim was to characterize the clinical and paraclinical presentations, therapeutic management, and outcomes in ... ...

Abstract	Background: Wilson's disease (WD) is usually diagnosed in children and young adults; limited data exist on late-onset forms. Objective: The aim was to characterize the clinical and paraclinical presentations, therapeutic management, and outcomes in patients with late-onset WD. Methods: Patients diagnosed with WD after age 40 years were identified from the French Wilson's Disease Registry (FWDR). Clinical, laboratory, and imaging findings and treatment were reported at diagnosis and last follow-up. Results: Forty-five patients were identified (median age: 49, range: 40-64) and placed in three groups according to their clinical presentation: neurological (n = 20, median diagnostic delay: 20 months), hepatic (n = 13, diagnostic delay: 12 months), and family screening (n = 12), all confirmed genetically. Six neurological patients had an atypical presentation (1 torticollis, 2 writer's cramps, 2 functional movement disorders, and 1 isolated dysarthria), without T2/fluid-attenuated inversion recovery brain magnetic resonance imaging (MRI) hyperintensities; 5 of 6 had no Kayser-Fleischer ring (KFR); 5 of 6 had liver involvement. In the neurological group, 84% of patients improved clinically, and 1 developed copper deficiency. In the hepatic group, 77% had cirrhosis; 6 patients required liver transplantation. In the screened group, 43% had mild liver involvement; 3 were not treated and remained stable; 24-h urinary copper excretion was normal in 33% of patients at diagnosis. Conclusions: In the FWDR, late-onset forms of WD affect 8% of patients, mostly with neurological presentations. Thirty percent of the neurological forms were atypical (isolated long-lasting symptoms, inconspicuous brain MRI, no KFR). With personalized treatment, prognosis was good. This study emphasized that WD should be suspected at any age and even in cases of atypical presentation. © 2022 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
MeSH term(s)	Adult ; Child ; Humans ; Middle Aged ; Young Adult ; Ceruloplasmin/metabolism ; Ceruloplasmin/therapeutic use ; Copper/metabolism ; Copper/therapeutic use ; Delayed Diagnosis ; Hepatolenticular Degeneration/diagnosis
Chemical Substances	Ceruloplasmin (EC 1.16.3.1) ; Copper (789U1901C5)
Language	English
Publishing date	2022-12-27
Publishing country	United States
Document type	Journal Article
ZDB-ID	607633-6
ISSN	1531-8257 ; 0885-3185
ISSN (online)	1531-8257
ISSN	0885-3185
DOI	10.1002/mds.29292
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Zs.A 2555: Show issues			Location: Je nach Verfügbarkeit (siehe Angabe bei Bestand) bis Jg. 1994: Bestellungen von Artikeln über das Online-Bestellformular Jg. 1995 - 2021: Lesesall (2.OG) ab Jg. 2022: Lesesaal (EG)
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Article ; Online: Encéphalopathie hépatique.

Jacques, Jérémie / Carrier, Paul / Debette-Gratien, Marilyne / Sobesky, Rodolphe / Loustaud-Ratti, Véronique

Presse medicale (Paris, France : 1983)

2016 Volume 45, Issue 1, Page(s) 46–59

Abstract: Hepatic encephalopathy is a severe complication of liver cirrhosis and is an important therapeutic challenge, with a social and economic issue. If, now, the pathophysiology is not totally understood (main role of ammonia, but a better understanding of ... ...

Title translation	Hepatic encephalopathy.
Abstract	Hepatic encephalopathy is a severe complication of liver cirrhosis and is an important therapeutic challenge, with a social and economic issue. If, now, the pathophysiology is not totally understood (main role of ammonia, but a better understanding of cerebral mechanisms), the clinical presentation is well-known. Some treatments are useful (disaccharides, treatment of the trigger) but their efficiency is limited. Nevertheless, the emergence of new treatments, such as non-absorbable antibiotics (rifaximin essentially), is an interesting therapeutic tool.
MeSH term(s)	Hepatic Encephalopathy/diagnosis ; Hepatic Encephalopathy/physiopathology ; Hepatic Encephalopathy/therapy ; Humans
Language	French
Publishing date	2016-01
Publishing country	France
Document type	English Abstract ; Journal Article
ZDB-ID	120943-7
ISSN	2213-0276 ; 0032-7867 ; 0755-4982 ; 0301-1518
ISSN (online)	2213-0276
ISSN	0032-7867 ; 0755-4982 ; 0301-1518
DOI	10.1016/j.lpm.2015.02.021
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Zs.A 794: Show issues			Location: Je nach Verfügbarkeit (siehe Angabe bei Bestand) bis Jg. 1994: Bestellungen von Artikeln über das Online-Bestellformular Jg. 1995 - 2021: Lesesall (1.OG) ab Jg. 2022: Lesesaal (EG)
Ua VI Zs.87: Show issues			Location: Je nach Verfügbarkeit (siehe Angabe bei Bestand) bis Jg. 2021: Bestellungen von Artikeln über das Online-Bestellformular ab Jg. 2022: Lesesaal (EG)

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Article ; Online: Post-Liver Transplantation Sinusoidal Obstruction Syndrome With Refractory Ascites Induced by Mycophenolate Mofetil.

Poli, Edoardo / Kounis, Ilias / Guettier, Catherine / Verstuyft, Céline / Coilly, Audrey / Sobesky, Rodolphe / Feray, Cyrille / Vibert, Eric / Ciacio, Oriana / Samuel, Didier / Bismuth, Henri / Duclos-Vallée, Jean-Charles

Hepatology (Baltimore, Md.)

2020 Volume 71, Issue 4, Page(s) 1508–1510

MeSH term(s)	Adult ; Anticoagulants/therapeutic use ; Ascites/chemically induced ; Ascites/drug therapy ; Hepatic Veno-Occlusive Disease/chemically induced ; Hepatic Veno-Occlusive Disease/drug therapy ; Humans ; Immunosuppressive Agents/administration & dosage ; Immunosuppressive Agents/adverse effects ; Liver Cirrhosis/surgery ; Liver Transplantation ; Male ; Mycophenolic Acid/administration & dosage ; Mycophenolic Acid/adverse effects
Chemical Substances	Anticoagulants ; Immunosuppressive Agents ; Mycophenolic Acid (HU9DX48N0T)
Language	English
Publishing date	2020-03-31
Publishing country	United States
Document type	Case Reports ; Journal Article
ZDB-ID	604603-4
ISSN	1527-3350 ; 0270-9139
ISSN (online)	1527-3350
ISSN	0270-9139
DOI	10.1002/hep.30984
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Article ; Online: Pediatric Wilson's Disease: Phenotypic, Genetic Characterization and Outcome of 182 Children in France.

Couchonnal, Eduardo / Lion-François, Laurence / Guillaud, Olivier / Habes, Dalila / Debray, Dominique / Lamireau, Thierry / Broué, Pierre / Fabre, Alexandre / Vanlemmens, Claire / Sobesky, Rodolphe / Gottrand, Frederic / Bridoux-Henno, Laure / Dumortier, Jérôme / Belmalih, Abdelouahed / Poujois, Aurelia / Jacquemin, Emmanuel / Brunet, Anne Sophie / Bost, Muriel / Lachaux, Alain

Journal of pediatric gastroenterology and nutrition

2021 Volume 73, Issue 4, Page(s) e80–e86

Abstract: Objectives: To describe a cohort of Wilson disease (WD) pediatric cases, and to point out the diagnostic particularities of this age group and the long-term outcome.: Methods: Clinical data of 182 pediatric patients included in the French WD national ...

Abstract	Objectives: To describe a cohort of Wilson disease (WD) pediatric cases, and to point out the diagnostic particularities of this age group and the long-term outcome. Methods: Clinical data of 182 pediatric patients included in the French WD national registry from 01/03/1995 to 01/06/2019 were gathered. Results: Diagnosis of WD was made at a mean age of 10.7 ± 4.2 years (range 1-18 years). At diagnosis, 154 patients (84.6%) had hepatic manifestations, 19 (10.4%) had neurological manifestations, and 9 patients (4.9%) were asymptomatic. The p.His1069Gln mutation was the most frequently encountered (14% of patients).Neurological patients were diagnosed at least 1 year after they presented their first symptoms. At diagnosis, the median urinary copper excretion (UCE) was 4.2 μmol/24 hours (0.2-253). The first-line treatment was d-penicillamine (DP) for 131 (72%) patients, zinc salts for 24 (13%) patients, and Trientine for 17 (9%) patients. Liver transplantation was performed in 39 (21.4%) patients, for hepatic indications in 33 of 39 patients or for neurological deterioration in 6 of 39 patients, mean Unified Wilson's Disease Rating Scale of the latter went from 90 ± 23.1 before liver transplantation (LT) to 26.8 ± 14.1 (P < 0.01) after a mean follow-up of 4.3 ± 2.5 years. Overall survival rate at 20 years of follow-up was 98%, patient and transplant-free combined survival was 84% at 20 years. Conclusion: Diagnosis of WD can be challenging in children, particularly at the early stages of liver disease and in case of neurological presentation; hence the support of clinical scores and genetic testing is essential. Diagnosis at early stages and proper treatment ensure excellent outcomes, subject to good long-term treatment compliance. LT is a valid option for end-stage liver disease not responding to treatment and can be discussed for selected cases of neurological deterioration.
MeSH term(s)	Adolescent ; Child ; Child, Preschool ; Copper ; France/epidemiology ; Hepatolenticular Degeneration/diagnosis ; Hepatolenticular Degeneration/genetics ; Hepatolenticular Degeneration/therapy ; Humans ; Infant ; Penicillamine/therapeutic use ; Retrospective Studies ; Treatment Outcome
Chemical Substances	Copper (789U1901C5) ; Penicillamine (GNN1DV99GX)
Language	English
Publishing date	2021-06-03
Publishing country	United States
Document type	Journal Article
ZDB-ID	603201-1
ISSN	1536-4801 ; 0277-2116
ISSN (online)	1536-4801
ISSN	0277-2116
DOI	10.1097/MPG.0000000000003196
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