Physicians planning to use penicillamine should thoroughly familiarize themselves with its toxicity, special dosage considerations, and therapeutic benefits. Penicillamine should never be used casually. Each patient should remain constantly under the close supervision of the physician. Patients should be warned to report promptly any symptoms suggesting toxicity.
Penicillamine is a chelating agent used in the treatment of Wilson's disease. It is also used to reduce cystine excretion in cystinuria and to treat patients with severe, active rheumatoid arthritis unresponsive to conventional therapy (see INDICATIONS).
CUPRIMINE is indicated in the treatment of Wilson's disease, cystinuria, and in patients with severe, active rheumatoid arthritis who have failed to respond to an adequate trial of conventional therapy. Available evidence suggests that CUPRIMINE is not of value in ankylosing spondylitis.
Published Studies Related to Cuprimine (Penicillamine)
Oral D-penicillamine for the prevention of retinopathy of prematurity in very low birth weight infants: a randomized, placebo-controlled trial. [2010.09]
PURPOSE: To compare prophylactic enteral D-penicillamine (DPA) with placebo for prevention of 'retinopathy of prematurity (ROP) or death' among very low birth weight (VLBW) infants... CONCLUSION: Prophylactic enterally administered DPA suspension in a dose 100 mg/kg/dose 8 h for 3 days, followed by 50 mg/kg once per day for next 11 days, does not prevent 'any stage ROP or death' or 'ROP requiring treatment' in VLBW infants. DPA is well tolerated and does not have any major short-term adverse effects. (c) 2010 The Author(s)/Journal Compilation (c) 2010 Foundation Acta Paediatrica.
Oral D-penicillamine for the prevention of retinopathy of prematurity in very low birth weight infants: a randomized, placebo-controlled trial. [2010.04.16]
Abstract Purpose: To compare prophylactic enteral D-penicillamine (DPA) with placebo for prevention of 'retinopathy of prematurity (ROP) or death' among very low birth weight (VLBW) infants. Methods: This was a double-blind, single-centre, randomized, placebo-controlled trial with stratification (for birth weight <1250 and >/=1250 g) and blocking...
High-dose versus low-dose D-penicillamine in early diffuse systemic sclerosis trial: lessons learned. [2004.02]
OBJECTIVES: To review important findings, or lessons, that were learned about measures of response, design, conduct, and analysis of a randomized, controlled trial (RCT), even though the trial failed to demonstrate efficacy of d-penicillamine... CONCLUSIONS: Even in studies that are therapeutically "negative," careful evaluation of the data can examine other hypotheses and thereby provide important insights into other aspects of trial design, outcome measures, patient function, and trial conduct.
The Disability Index of the Health Assessment Questionnaire is a predictor and correlate of outcome in the high-dose versus low-dose penicillamine in systemic sclerosis trial. [2001.03]
OBJECTIVE: To explore the clinical implications of a score of > or =1.0 on the Disability Index of the Health Assessment Questionnaire (HAQ DI) at the first patient visit, and to examine the implications of improvement in HAQ DI score over 2 years in a cohort of systemic sclerosis (SSc) patients with diffuse cutaneous scleroderma... CONCLUSION: A baseline HAQ DI score of > or =1.0 predicted mortality over 4 years. Improvement in the HAQ DI score in these patients with diffuse scleroderma was associated with improvement in skin thickening, hand function, oral aperture, lung function, signs of arthritis, serum creatinine level, and the investigator's global assessment of improvement. The HAQ DI is a self-administered questionnaire that SSc patients can complete easily and rapidly and that gives the practicing physician important information about prognosis, patient status, and changes in disease course over time.
D-penicillamine is not an effective treatment in systemic sclerosis. 
Based on open studies. D-penicillamine (DPA) has been used for the treatment of systemic sclerosis (SSc) but we believe the controlled trial of this drug in SSc does not support its use to treat this disease.
Clinical Trials Related to Cuprimine (Penicillamine)
Penicillamine Chelation for Children With Lead Poisoning [Active, not recruiting]
Childhood Lead Poisoning is a widespread disease that has few effective treatments. The
specific aims of this proposed clinical trial are threefold:
- To determine whether a six-week course of a newly formulated d-penicillamine suspension
will effectively reduce blood lead level in children aged 6 months to 16 years with
blood lead levels of 15-25 μg/dL.
- To determine whether d-penicillamine chelation produces a sustained reduction in blood
lead level in comparison with succimer and other lead chelators which always produce a
significant post-treatment "rebound".
- To determine whether chelation with d-penicillamine improves the physiologic
disturbances that can be measured in children with blood lead levels in this range.
Penicillamine, Low Copper Diet, and Radiation Therapy in Treating Patients With Glioblastoma [Active, not recruiting]
RATIONALE: Penicillamine may stop the growth of glioblastomas by stopping blood flow to the
tumor. A diet low in copper may interfere with the growth of brain tumor cells. Radiation
therapy uses high-energy x-rays to damage tumor cells. Combining these therapies may be
effective in treating glioblastoma.
PURPOSE: Phase II trial to study the effectiveness of penicillamine, a low copper diet, and
radiation therapy in treating patients who have newly diagnosed glioblastoma.
Study of Positron Emission Tomography and Computed Tomography in Guiding Radiation Therapy in Patients With Stage III Non-Small Cell Lung Cancer [Recruiting]
RATIONALE: Imaging procedures, such as positron emission tomography (PET) scan and CT scan,
may help doctors plan radiation therapy for patients with non-small cell lung cancer.
PURPOSE: This randomized phase II trial studies how well PET/CT scan work in guiding
radiation therapy compared to standard radiation therapy treatment in patients with stage
III non-small cell lung cancer.
Efficacy of Invitro Expanded Bone Marrow Derived Allogeneic Mesenchymal Stem Cell Transplantation Via Portal Vein or Hepatic Artery or Peripheral Vein in Patients With Wilson Cirrhosis [Recruiting]
Wilson's disease is an autosomal recessive genetically inherited disorder of copper
metabolism, causing neurological, psychiatric and liver disease. The ATP7B gene on the 13th
chromosome is responsible for the disease. Liver has a critical role on copper metabolism.
It is the main site of copper accumulation and bile secretion is the only physiologic way of
copper elimination. Due to defective production of ceruloplasmin which carries copper, wide
amount of free copper precipitates throughout the body but particularly in the liver, eyes
and brain. Patients are bound to lifelong chelating agents such as penicillamine, trientine
and tetramine dihydrochloride. Unfortunately, these medications may cause severe
side-effects such as hypersensitivity reactions, bone marrow suppression, auto-immune
disease and sideroblastic anemia. Medical treatment of liver cirrhosis, the last stage of
the illness that leads to morbidity and mortality in the Wilson Disease, is difficult. Liver
transplantation is still the most effective treatment for the patients with liver cirrhosis
in Wilson Disease. However, serious problems are accompanied with liver transplantation.
Lack of liver donors, complications during and after the surgery, graft rejection and high
costs are the main problems.
There are cells in the human body that are capable to renew themselves and differentiate to
a diverse range of specialized cell types. These are called "stem cells". Stem cells can be
differentiated to specialized cells in appropriate medias in the laboratory. Recently, the
differentiation potential of mesenchymal stem cells into hepatocytes is proved by
demonstrating hepatocytes containing Y chromosome in the female who has had bone marrow
transplantation from male donors. In many laboratory studies, it is observed that human bone
marrow derived mesenchymal stem cells, transplanted to animals with induced liver damage,
differentiate into the albumin producing hepatocytes without fusion. By these studies, it is
understood that mesenchymal stem cells are more potent than other bone marrow elements in
context of differentiation to hepatocytes. Even though the number of studies on human for
the same purpose is few, findings are supporting those of animal experiments. Mesenchymal
stem cells are non-immunogenic. Safety and feasibility of allogeneic transplantations
between individuals without need of immunosuppressive drug regimen are proven. Proofs of
correcting metabolic defects by this way are also presented in some publications. For the
reasons mentioned above, allogeneic mesenchymal stem cell transplantation is a promising
treatment modality especially for the hereditary metabolic diseases. By this way,
non-immunogeneic mesenchymal stem cells which have healthy genetic structure, can
manufacture the required enzyme, will be repopulated in the damaged tissue and contribute to
the clinical improvement.
In this study, mesenchymal stem cells will be derived from healthy volunteer donor's bone
marrow and be expanded in-vitro, and then 1 million cells per kg will be infused to patients
with liver cirrhosis related to Wilson disease, 50 million cells via hepatic artery and the
remaining cells via peripheral vein. It is aimed to enable liver regeneration, decrease
fibrosis rate, improve patient's health conditions, increase ceruloplasmin synthesis,
ameliorate disorder of copper metabolism, decrease the need for chelating agents, increase
living standards of patients, and prolong waiting time for liver transplantation. Finally it
is aimed to establish a new and regenerative treatment protocol alternative to liver
transplantation. For observation of clinical and laboratory improvement, patients are
planned to be monitored by histopathologic examination of liver biopsies before and at 6th
month after the treatment, monthly biochemical and hematologic blood tests and periodic
radiologic examinations. This is a hopeful, avant garde and sophisticated study which may
constitute new horizons in context of cellular therapies.
Page last updated: 2011-12-09