Adverse Events with d-penicillamine Therapy in Hepatic Wilson’s Disease: A Single-Center Retrospective Audit

Kumar, Sanjay; Patra, Biswa Ranjan; Irtaza, Mohammed; Rao, Praveen; Giri, Suprabhat; Darak, Harish; Gopan, Amrit; Kale, Aditya; Shukla, Akash

doi:10.1007/s40261-022-01117-x

Cited by 9 publications

(2 citation statements)

References 27 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…9 In a single center retrospective audit, penicillamine also caused significant hematologic and renal adverse events, with significant intolerance noted in 28 of the 112 patients studied. 10 There is an ongoing phase 3 study for an adeno-associated virus-mediated gene transfer therapy for WD, which depending on the outcome could change this therapeutic conversation drastically. 11 This study is planned to complete in 2031.…”

Section: Discussionmentioning

confidence: 99%

Treating the Neurologic Manifestations of Wilson’s Disease With Liver Transplantation

Marmol,

Maeder-York,

Hislop

et al. 2023

The Neurohospitalist

View full text Add to dashboard Cite

Wilson’s Disease (WD) manifests with systemic and neuropsychiatric symptoms, caused by an ATP7B genetic mutation, leading to an accumulation of copper. Presentations are diverse and the diagnosis should be considered in anyone under 50 with a new onset movement disorder. Early recognition and treatment can limit morbidity. While liver transplantation (LT) is recommended in WD patients with hepatic failure, its use for pure neurologic indication remains controversial. We present a patient who failed medical management and underwent LT for pure neurologic indications. Subsequent neurologic symptom improvement supports the use of LT for patients with pure neurologic manifestations of WD.

show abstract

Section: Discussionmentioning

confidence: 99%

Treating the Neurologic Manifestations of Wilson’s Disease With Liver Transplantation

Marmol,

Maeder-York,

Hislop

et al. 2023

The Neurohospitalist

View full text Add to dashboard Cite

show abstract

“…Unfortunately, PSH may give rise to adverse effects [ 89 ], the most serious being immune reactions leading, e.g., to immune complex nephritis. Furthermore, penicillamine may exacerbate neurological symptoms in the initial phase of treatment [ 90 ]. Another water-soluble chelator, triethylene tetramine (Trien), has been recommended as an alternative drug [ 91 ].…”

Section: Chelator Combination Versus Monotherapy As Therapeutic Strategymentioning

confidence: 99%

Chelation Combination—A Strategy to Mitigate the Neurotoxicity of Manganese, Iron, and Copper?

Aaseth

Nurchi

2022

Biomolecules

View full text Add to dashboard Cite

The chelating thiol dimercaptosuccinate (DMSA) and the traditional agent D-penicillamine (PSH) are effective in enhancing the urinary excretion of copper (Cu) and lead (Pb) in poisoned individuals. However, DMSA, PSH, EDTA (ethylenediamine tetraacetate), and deferoxamine (DFOA) are water-soluble agents with limited access to the central nervous system (CNS). Strategies for mobilization of metals such as manganese (Mn), iron (Fe), and Cu from brain deposits may require the combined use of two agents: one water-soluble agent to remove circulating metal into urine, in addition to an adjuvant shuttler to facilitate the brain-to-blood mobilization. The present review discusses the chemical basis of metal chelation and the ligand exchange of metal ions. To obtain increased excretion of Mn, Cu, and Fe, early experiences showed promising results for CaEDTA, PSH, and DFOA, respectively. Recent experiments have indicated that p-amino salicylate (PAS) plus CaEDTA may be a useful combination to remove Mn from binding sites in CNS, while the deferasirox–DFOA and the tetrathiomolybdate–DMSA combinations may be preferable to promote mobilization of Fe and Cu, respectively, from the CNS. Further research is requested to explore benefits of chelator combinations.

show abstract

Early neurological deterioration in Wilson’s disease: a systematic literature review and meta-analysis

et al. 2023

View full text Add to dashboard Cite

Introduction Neurological deterioration, soon after anti-copper treatment initiation, is problematic in the management of Wilson’s disease (WD) and yet reports in the literature are limited. The aim of our study was to systematically assess the data according to early neurological deteriorations in WD, its outcome and risk factors. Methods Using PRISMA guidelines, a systematic review of available data on early neurological deteriorations was performed by searching the PubMed database and reference lists. Random effects meta-analytic models summarized cases of neurological deterioration by disease phenotype. Results Across the 32 included articles, 217 cases of early neurological deterioration occurred in 1512 WD patients (frequency 14.3%), most commonly in patients with neurological WD (21.8%; 167/763), rarely in hepatic disease (1.3%; 5/377), and with no cases among asymptomatic individuals. Most neurological deterioration occurred in patients treated with d-penicillamine (70.5%; 153/217), trientine (14.2%; 31/217) or zinc salts (6.9%; 15/217); the data did not allow to determine if that reflects how often treatments were chosen as first line therapy or if the risk of deterioration differed with therapy. Symptoms completely resolved in 24.2% of patients (31/128), resolved partially in 27.3% (35/128), did not improve in 39.8% (51/128), with 11 patients lost to follow-up. Conclusions Given its occurrence in up to 21.8% of patients with neurological WD in this meta-analysis of small studies, there is a need for further investigations to distinguish the natural time course of WD from treatment-related early deterioration and to develop a standard definition for treatment-induced effects.

show abstract

Adverse Events with d-penicillamine Therapy in Hepatic Wilson’s Disease: A Single-Center Retrospective Audit

Cited by 9 publications

References 27 publications

Treating the Neurologic Manifestations of Wilson’s Disease With Liver Transplantation

Treating the Neurologic Manifestations of Wilson’s Disease With Liver Transplantation

Chelation Combination—A Strategy to Mitigate the Neurotoxicity of Manganese, Iron, and Copper?

Early neurological deterioration in Wilson’s disease: a systematic literature review and meta-analysis

Contact Info

Product

Resources

About