Orfadin and Hepatic neoplasm - a phase IV clinical study of FDA data

Summary:

Hepatic neoplasm is reported as a side effect among people who take Orfadin (nitisinone), especially for people who are male, 2-9 old, have been taking the drug for 1 - 6 months also take Spironolactone, and have Tyrosinaemia.

The phase IV clinical study analyzes which people have Hepatic neoplasm when taking Orfadin. It is created by eHealthMe based on reports of 968 people who have side effects when taking Orfadin from the FDA, and is updated regularly.

What is Orfadin?

Orfadin has active ingredients of nitisinone. eHealthMe is studying from 968 Orfadin users. Check the latest studies of Orfadin.

What is Hepatic neoplasm?

Hepatic neoplasm (liver tumour) is found to be associated with 490 drugs and 519 conditions by eHealthMe. Check the latest studies of Hepatic neoplasm.

eHealthMe: drug outcomes in the real world

eHealthMe runs one of the largest post-marketing drug safety studies in the world. We study millions of patients and 5,000 more each day. Our data-driven phase IV clinical trials have been referenced on 800+ peer-reviewed medical publications including The Lancet, Mayo Clinic Proceedings, and Nature. Tools to study our phase IV findings are available to the public, anonymous and free >>>.

On Aug, 31, 2026

968 people reported to have side effects when taking Orfadin.
Among them, 18 people (1.86%) have Hepatic neoplasm.

Among these 18 people:

How long have people been on Orfadin when they have Hepatic neoplasm? *

What is the gender of people who have Hepatic neoplasm when taking Orfadin? *

female: 33.33 %
male: 66.67 %

What is the age of people who have Hepatic neoplasm when taking Orfadin? *

What are other drugs people take besides Orfadin? *

Spironolactone: 1 person, 5.56%

What are other side effects people have besides Hepatic neoplasm? *

Hepatic Cirrhosis (chronic liver disease characterized by replacement of liver tissue by fibrosis, scar tissue): 5 people, 27.78%
Alpha 1 Foetoprotein Increased: 3 people, 16.67%
Thrombocytopenia (decrease of platelets in blood): 1 person, 5.56%
Tachypnea: 1 person, 5.56%
Breathing - Slowed Or Stopped: 1 person, 5.56%
Cytomegalovirus Infection: 1 person, 5.56%
Diaphragmatic Paralysis (acquired dysfunction of the phrenic nerve): 1 person, 5.56%
Drug Ineffective: 1 person, 5.56%
Haemoglobin Decreased: 1 person, 5.56%
Hepatic Dysplasia (enlargement of liver): 1 person, 5.56%

What are the existing conditions these people have? *

Tyrosinaemia (tyrosine metabolism resulting in liver and kidney disturbances and mental retardation): 15 people, 83.33%

* Approximation only. Some reports may have incomplete information.

Do you take Orfadin and have Hepatic neoplasm?

- Check whether Hepatic neoplasm is associated with a drug or a condition
- Predict drug outcomes for up to one year with AI
- Get an AI agent to monitor your drugs continuously

Related studies:

Effectiveness of, long term effects of, and alternative drugs to Orfadin:

Orfadin (968 reports)

Browse all side effects of Orfadin:

a b c d e f g h i j k l m n o p q r s t u v w x y z

Browse all the drugs that are associated with Hepatic neoplasm:

Hepatic neoplasm (490 drugs)

Browse all the conditions that are associated with Hepatic neoplasm:

Hepatic neoplasm (519 conditions)

How the study uses the data?

The study uses data from the FDA. It is based on nitisinone (the active ingredients of Orfadin) and Orfadin (the brand name). Other drugs that have the same active ingredients (e.g. generic drugs) are not considered. Dosage of drugs is not considered in the study.

How to use the study?

DO NOT STOP MEDICATIONS without first consulting your doctor. If there are any serious or long term adverse effects discovered in the study, discuss the study with your doctor to ensure that proper medication management will be in place if applicable.

Who is eHealthMe?

With medical big data and proven AI/ML algorithms, eHealthMe provides a platform for everyone to run phase IV clinical trials. We study millions of patients and 5,000 more each day. Results of our real-world drug study have been referenced on 800+ peer-reviewed medical publications, including The Lancet, Mayo Clinic Proceedings, and Nature. Our analysis results are available to researchers, health care professionals, patients (testimonials), and software developers (open API).

WARNING, DISCLAIMER, USE FOR PUBLICATION

WARNING: Please DO NOT STOP MEDICATIONS without first consulting a physician since doing so could be hazardous to your health.

DISCLAIMER: All material available on eHealthMe.com is for informational purposes only, and is not a substitute for medical advice, diagnosis, or treatment provided by a qualified healthcare provider. All information is observation-only. Our phase IV clinical studies alone cannot establish cause-effect relationship. Different individuals may respond to medication in different ways. Every effort has been made to ensure that all information is accurate, up-to-date, and complete, but no guarantee is made to that effect. The use of the eHealthMe site and its content is at your own risk.

If you use this eHealthMe study on publication, please acknowledge it with a citation: study title, URL, accessed date.