EXPLorative Data Collection for Patient chAracterIzation, treatmeNt Pathways and Outcomes of IRON Preparations (EXPLAIN-IRON)

By describing the characteristics of iron deficiency (ID) patients treated with various oral or intravenous iron formulations and their outcomes, this registry will provide the medical community with important information to support treatment decisions for their patients regarding data on effectiveness, safety, tolerability, treatment persistence, quality of life, and therapeutic costs. This will ultimately support improvements to patient care, including the long-term outcomes of patients with ID.

Study Overview

• Status: Completed
• Conditions: Iron-deficiency
• Intervention / Treatment: Drug: Iron Supplement

Detailed Description

Iron deficiency (ID) anaemia is a prevalent condition in Germany and other Western countries. It is a common complication in inflammatory bowel disease (IBD), cardiovascular disease including chronic heart failure, cancer, chronic kidney disease (CKD), gynaecological conditions, and others.

Anaemia has a strong impact on patient's quality of life and ability to work, and removing the condition by increasing the haemoglobin may improve QoL and other patient-related outcomes. While in certain patient groups, such as CKD, anaemia is addressed on a routine basis, in others it remains widely undertreated. For example, in IBD patients across various European countries, iron supplementation was administered in only up to 28% of individuals.

For iron supplementation aimed to replenish to body' iron stores, there are numerous oral, and intravenous preparations available.

The most convenient approach is oral administration, where Fe2+ salts are mostly used. Usually low doses between 50 - 100 mg daily are recommended, as the duodenum can only absorb 10 - 20 mg daily, and higher doses are associated with gastrointestinal side effects including diarrhoea, nausea, flatulence and gastric erosions. To reduce these common side effects, Fe3+ formulations like ferric maltol (Feraccru) have been newly introduced.

Intravenous preparations are all Fe3+ oxyhydroxides with a carbohydrate coat (iron dextran, gluconate, sucrose, carboxymaltose, or ferumexytol). These preparations are typically used second-line in patients with ID who had unsatisfactory treatment results under oral treatment. IV iron infusions have been associated with hypophosphataemia and hypersensitivity reactions. However these events depend on the preparation and are infrequent.

EXPLAIN-IRON has been set up as the first interdisciplinary registry to add further information on the situation and management of patients who receive iron substitution in various indications: IBD, chronic kidney disease, cancer- or therapy-induced ID, gynaecological conditions (hypermenorrhoea, post-partum, fatigue), cardiological indications (congenital heart disease; chronic heart failure) and other causes of ID.

The registry will allow for documentation of all approved oral and IV preparations. As the registry also serves the specific purpose to collect data on the newly introduced preparation Feraccru (oral ferric maltol), about half of the patient population will be treated with that agent.

The registry will be of interest to assess to which extent the various guidelines on iron substitution have been adopted in clinical practice. Overall, EXPLAIN-IRON is expected to provide a comprehensive picture on the use and the outcomes of iron substitution in Germany. By describing the characteristics of ID patients treated with various iron formulations and their outcomes, this registry will provide the medical community with important information to support treatment decisions for their patients regarding data on effectiveness, safety, tolerability, treatment persistence, quality of life, and therapeutic costs. This will ultimately support improvements to patient care, including the long-term outcomes of patients with ID.

• Study Type: Observational
• Enrollment (Actual): 51

Contacts and Locations

Study Locations

• Germany
  • Berlin, Germany, 10318
    • Praxis fur Gastroenterologie
  • Kiel, Germany, 24105
    • Klinik für Innere Medizin I des Universiätsklinikums

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 99 years (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Patients with iron deficiency in one of the following indications:

(i) Inflammatory bower disease, (ii) chronic kidney disease, (iii) cancer- or therapy-induced iron deficiency, (iv) gynaecological conditions (hypermenorrhea, post-partum, fatigue), (v) cardiology (including iron deficiency in patients with congenital heart disease or chronic heart failure), and (vi) diverse other causes of iron deficiency.

Description

Inclusion Criteria:

• Female or male patients, aged at least 18 years
• Clinical diagnosis of iron deficiency according to treating physician
• Decision to treat with an oral or intravenous iron supplementation made by treating physician considering the indications of the respective Summary of Product Characteristics (prescribing information)
• maintenance or newly initiated treatment (including returning quitters)
• written informed consent

Exclusion Criteria:

• patient not available for long-term documentation
• concomitant or planned participation in a clinical trial (on iron supplementation)
• concomitant over-the-counter iron supplementation

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Hemoglobin	serum level change compared to baseline	at 3 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Ferritin	serum level change compared to baseline	at 3 months
Hemoglobin	time to normalisation	2 years
Ferritin	time to normalisation	2 years

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Transferrin saturation	Time to normalisation	at 3 months
Adverse events	Type, severity and time of adverse events that are related to and caused by iron treatment (in the physician's opinion)	2 years
Hypophosphatemia	Percentage of patients	2 years
Self-assessed Quality of life	course over time, by Euroquol five-dimensional questionnaire	2 years
Fatigue	course over time, by fatigue-specific FACIT questionnaire	2 years
Discontinuation of iron supplementation	Rate of patients	2 years
Economic parameters	costs for days of hospitalisation and for physician contacts	2 years
Patient satisfaction	Patients' Treatment Satisfaction for Medication (TSQM-9 questionnaire)	2 years

Collaborators and Investigators

• Sponsor: GWT-TUD GmbH
• Collaborators: Shield Therapeutics
• Investigators: Study Director: David Pittrow, MD, PhD, GWT-TUD GmbH; Principal Investigator: Stefan Schreiber, MD, PhD, Klinik für Innere Medizin I, Universitätsklinikum Kiel

Study record dates

Study Major Dates

• Study Start (ACTUAL): January 16, 2018
• Primary Completion (ACTUAL): April 20, 2019
• Study Completion (ACTUAL): April 20, 2019

Study Registration Dates

• First Submitted: December 12, 2017
• First Submitted That Met QC Criteria: December 18, 2017
• First Posted (ACTUAL): December 22, 2017

Study Record Updates

• Last Update Posted (ACTUAL): July 2, 2019
• Last Update Submitted That Met QC Criteria: July 1, 2019
• Last Verified: July 1, 2019

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Hematologic Diseases; Anemia, Hypochromic; Anemia; Iron Metabolism Disorders; Anemia, Iron-Deficiency; Physiological Effects of Drugs; Trace Elements; Micronutrients; Hematinics; Iron; Ferric maltol
• Other Study ID Numbers: EXPLAIN IRON

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No