To examine if intravenous administration of iron ismore efficacious than oral iron in improvement offitness scores, iron status and reduction of fatigue
ID
Source
Brief title
Condition
- Gastrointestinal inflammatory conditions
- Autoimmune disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
8.1.1 Main study parameter/outcome
Primary outcome is the proportion of patients per group that show a 15%
increase in 6 minute walking distance from study baseline.
The six minute walktest (6MWT) is an established method to assess exercise
capacity. It is expressed as the distance a person can walk at a constant,
uninterrupted pace in 6 minutes {2002 263 /id}. The patients are asked to walk
up and down the measured lap at their best pace but not to run or race. With
patients tested by the same technician, short-term reproducibility of the 6MWT
is excellent {2002 263 /id}. 6MWT is expressed as absolute distance in meters
and is age and height dependent {Geiger, 2007 557 /id;Lammers, 2008 558 /id}.
Secondary outcome
8.1.2 Secondary outcome variables:
Secondary outcome variable in the study is an increase of Hb with 1.25 mmol/L
(2 g/dl) one month after administration of IV ferric carboxymaltose therapy
compared to the Hb level at time of inclusion
Other secondary endpoints include the IMPACT-III score and the PEDSQL fatigue
scale.
The IMPACT-III score is a disease-specific quality
of life score, composed of 35 items on 6 domains: IBD-related symptoms (7
items),
systemic symptoms (3), emotional functioning (7), social functioning (12), body
image (3) and treatment/intervention-related concerns (3).[26,27] Each item can
be
scored on a 5 point Likert scale, coded from 0 to 4 points. Higher scores
indicate
better quality of life. The IMPACT questionnaire is validated for use in
children 8
years and older and is recommended for the evaluation of new therapies because
of
its high sensitivity to change. The IMPACT-III (NL) is a translated and modified
version of the original Canadian questionnaire that used a visual analogue
scale.[28,29] The Likert scale was introduced as it has been shown that children
consider it easier to complete than a visual analogue scale.
The PEDSQL fatigue scale: The developed 18-item PedsQL Multidimensional Fatigue
Scale was designed to measure fatigue in pediatric patients and comprises the
General Fatigue Scale (6 items), Sleep/Rest Fatigue Scale (6 items), and
Cognitive Fatigue Scale (6 items). (Varni, 2004) The questionnaire is available
in Dutch for children (8-12y) and adolescents (12-18y). The questionnaire
comprises parallel child self-reports and parent proxy-reports. The
participants rate how often a particular problem occurred in the past month,
using a 5-point Likert scale. Each item is reverse-scored and rescaled to 0-100
scale, so that higher scores indicate fewer symptoms of fatigue.
Also monitored parameters are the clinical disease activity according to PCDAI
and PUCAI,
laboratory markers for effectiveness of IV iron therapy in replenishment of
iron stores/biomarkers for iron stores (Ht, cell indices, thrombocytes,
ferritin, transferrin, serum iron level, transferrin saturation, reticulocytes
, sTfR, CRP, soluble transferrin receptors to log ferritin (sTfR-F ratio),
transferrin/log ferritin ratio, hepcidin), side effects of IV iron therapy on
liver functioning (AST, ALT, AF, total protein, albumin) and side effects on
electrolyte homeostasis (phosphate).
Background summary
Inflammatory bowel disease (IBD) in children and adolescents is often
associated with anemia. Anemia in IBD has multiple
etiologies and there is often a combination of iron
At the time that children are diagnosed with inflammatory bowel disease (IBD)
over 80% of them are anemic with iron deficiency being the most common cause.
Despite reaching remission, fatigue and decreased physical fitness continue to
disturb activities of daily living. For children, this can be the most
debilitating aspect of their disease.
In order to replete empty iron stores administration of iron is recommended,
yet the preferred route of administration has not been determined in children.
Data about efficacy and optimal timing of IV and oral iron administration in
pediatric patients are lacking.
We hypothesize that children with IDA
receiving IV iron therapy in comparison to oral iron
therapy will have faster recovery from anemia in
terms of Hb and other hematologic parameters and
perform better in tests for fitness and score lower in fatigue scales.
Study objective
To examine if intravenous administration of iron is
more efficacious than oral iron in improvement of
fitness scores, iron status and reduction of fatigue
Study design
Prospective, multicentre randomised clinical trial
comparing IV and oral iron administration in
children and adolescents with IBD
Intervention
Intravenous administration of ferric carboxymaltose (intervention):
The study medication is ferric carboxymaltose or ijzercarboxymaltose in Dutch,
brand name Ferinject ® (The Netherlands) or Injectafer® (Belgium) . Intravenous
ferric carboxymaltose has several potential benefits in the treatment of anemia
in children with IBD over oral therapy or other intravenous formulations.
Studies performed in both adult and pediatric populations indicate a faster
correction of anemia, a more profound replenishment of iron stores and fewer
side effects compared to oral supplementation (8,9,10). The benefit of ferric
carboxymaltose over other intravenous formulations is the possibility to safely
treat anemia in a single intravenous dose. Especially in children this is more
patient-friendly.
Usual care
Standard medical treatment is provided to the patients as an oral iron
supplement (ferrofumarate), dosing will be 9 mg/kg/day in 2 doses with a
maximum of 600 mg (according to a frequently used dutch formulary,
www.kinderformularium.nl).
Medication will be provided as a suspension or as a tablet, depending on the
preference of the patient and the possibility to reach the correct dose when
using tablets.
Study burden and risks
In the intervention group, risks attributed to the intravenous iron therapy are
hypersensitivity reactions, headache and dizziness and with paravenous leakage
brown discolouration and irritation of the skin may occur.
The number of venapunctures for sampling is the same for both groups and is not
different from routine patient care. However, collection of stool samples and
completion of questionnaires needs to be done periodically. The 6 minute
walking test will be done at baseline, 4- and 12 weeks later and 6 months after
start of study.
Benefit in the intervention group is that patients will have more information
about their disease state, laboratory values and fitness.
Henri Dunantstraat 5
Heerlen 6419 PC
NL
Henri Dunantstraat 5
Heerlen 6419 PC
NL
Listed location countries
Age
Inclusion criteria
*1. Children attending a pediatrician/ pediatric gastro-enterologist/gastro-enterologist.
2. Children aged 8 * 20 years
3. Suffering from CD/CU/IBDU diagnosed according to the Porto criteria (1)
4. Written informed consent of both parents with authority or from custodial parent and if age> 12y: also from the child itself.
5. Ability to understand and speak Dutch language.
Exclusion criteria
1. Allergic reactions to intravenous iron therapy
2. Suffering from hemochromatosis or other iron overload disease
3. Patients who received oral/ intravenous iron therapy three months prior to the study
4. PUCAI > 65 - PCDAI > 30 (severe disease activity)
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2012-005644-26-NL |
| CCMO | NL42995.096.12 |
| OMON | NL-OMON27310 |