EAL

TN: Telenutrition Interventions by Registered Dietitians (2012)

Citation:

Study Design:

Class:

- Click here for explanation of classification scheme.

Quality Rating:

Research Purpose:

To study the effects of online availability of individual Phe (phenylalanine) levels to PKU (phenylketonuria) patients and families without concurrent instruction on patient safety, plasma Phe levels, frequency of blood sampling and satisfaction.

Inclusion Criteria:

Patients diagnosed with PKU at newborn screening and continuously-treated with protein-restricted diet and supplementation of amino acids
Patients with classical PKU (untreated Phe level over 1,000mcmol per L, Phe tolerance under 500mg per day)
Patients with non-PKU hyperphenylalaninemia or non-PKU HPA (untreated Phe level over 1,000mcmol per L, Phe tolerance over 500mg per day).

Exclusion Criteria:

Patients less than one year of age
No access to internet at home
Pregnant or the desire to become pregnant during the study period.

Description of Study Protocol:

Recruitment

Participants were recruited by treating physicians in the participating metabolic centers.

Design

Randomized controlled trial
Patients randomly-assigned to one of two groups (Group One–Intervention; Group Two–Control)
Randomization performed in a 1:1 ratio by TENALEA Clinical Trial Data Management System, using non-deterministic minimization method.

Blinding Used

Implied with measurements.

Intervention

Group One (Intervention Group)
- Given access to a secured personal web page called "My PKU"
- Parents and patients were emailed when results were available
- Web page contained graphs of trend in Phe values over last 12 months, links to dietary guidance and causes of elevated Phe, as well as suggested actions to correct values to advised range
- Changes in supplements were made only with consultation with the dietitian
- The dietitian could be contacted via the web page and questions were answered within three days.
Group Two (Control Group)
- Received standard of care, receiving a phone call by the dietitian if the Phe value was out of the advised range
- They could also receive the value via phone or email.
Parents of children less than 18 years of age received the information or were given access to "My PKU" and were encouraged to review the information with the child.

Statistical Analysis

Used SPSS 16.0
Mann-Whitney tests were used to detect differences between both groups for age, mean Phe value, sample frequency and the percentage Phe vales above the recommended range
Wilcoxon signed-rank tests were used to determine differences between both study periods, within each group.

Data Collection Summary:

Timing of Measurements

Study period lasted 10 weeks. Patients knew of the recommended blood sampling frequencies by age.
Pre-study period used for comparison was 10 weeks before study began
Group One had a sub-group analysis performed for age groups under 10 years old and 10 years and up (because PKU patients are more vulnerable to high Phe levels at a younger age)
Patient satisfaction of Intervention Group was assessed six months after the study period.

Dependent Variables

Median Phe value (mcmol per L) pre-study period (10 weeks) compared to study period (10 weeks)
Percent Phe values above recommended range pre-study and during study period
Median sample frequency (range) in 10 months (pre-study and study periods compared)
Whether and within how many hours of receiving the email that a new Phe value was available did the patients or parents log on to 'My PKU' (website reported these measures)
Patient satisfaction for Intervention Group (Group One) was measured six months after the study period via email.

Independent Variables

Group One (Intervention Group)
- Given access to a secured personal web page called "My PKU"
- Parents or patients were emailed when results were available
- Web page contained graphs of trend in Phe values over last 12 months, links to dietary guidance and causes of elevated Phe, as well as suggested actions to correct values to advised range
- Changes in supplements were made only with consultation with the dietitian
- The dietitian could be contacted via the web page and questions were answered within three days.
Group Two (Control Group)
- Received standard of care, receiving a phone call by the dietitian if the Phe value was out of the advised range
- They could also receive the value via phone or email.

Control Variables

Sex
Age.

Description of Actual Data Sample:

Initial N

68 (24 adults, 44 children) were eligible and invited to participate
38 patients (11 adults) consented and were randomized to either Group One (22 patients) or Group Two (16 patients).

Attrition (Final N)

37
Group One had seven males and 14 females
Group Two had seven males and nine females
One patient became pregnant and was excluded; all others completed the study.

Age

Group One: Mean, 15±9.5 years; range, one to 35
Group Two: Mean, 10.9±8.6 years; range, one to 28.

Ethnicity

Not reported.

Other Relevant Demographics

Group One: 16 patients with classic PKU and five with non-PKU HPA
Group Two: Nine patients with classic PKU and seven patients with non-PKU HPA.

Anthropometrics

Both groups were comparable for age and sex and study participants were comparable to non-participants for age and sex.

Location

Amsterdam, the Netherlands.

Summary of Results:

Key Findings

No statistically-significant difference between pre-study and study period mean Phe value for either group
No statistically-significant difference between pre-study and study period percent Phe values above recommended levels for either group
No statistically-significant difference in sub-group analysis of group for median Phe values or percent Phe values above recommended levels for Group One
No statistically-significant difference in sample frequency pre-study or during study period for either group
100% of patients or parents in Group One (Intervention Group) responded highly positively to patient satisfaction question
81% of Phe values were checked within 12 hours of posting and 88% within 48 hours
5.5% (N=11) were not viewed before a new Phe value was entered (of the 11, nine patients had an appointment with the dietitian the day the new result was available and the dietitian had given them the value).

Author Conclusion:

Providing online Phe values to PKU patients is feasible, not harmful and is well-accepted by patients and families.

Funding Source:

Industry:

Nutricia

Other:

In-Kind support reported by Industry:

Yes

Reviewer Comments:

Relatively small sample size and groups were not similarly sized
All participants had to have access to the Internet and may not be representative of the PKU population
The "MyPKU" tool was built by Nutricia, which also provided study funding
No measures were found to be statistically significant, but statistical analysis was not very robust
Measure of satisfaction was not compared across groups.

Quality Criteria Checklist: Primary Research
Relevance Questions
	1.	Would implementing the studied intervention or procedure (if found successful) result in improved outcomes for the patients/clients/population group? (Not Applicable for some epidemiological studies)	Yes
	2.	Did the authors study an outcome (dependent variable) or topic that the patients/clients/population group would care about?	Yes
	3.	Is the focus of the intervention or procedure (independent variable) or topic of study a common issue of concern to dieteticspractice?	Yes
	4.	Is the intervention or procedure feasible? (NA for some epidemiological studies)	Yes

Validity Questions
1.	Was the research question clearly stated?		Yes
	1.1.	Was (were) the specific intervention(s) or procedure(s) [independent variable(s)] identified?	Yes
	1.2.	Was (were) the outcome(s) [dependent variable(s)] clearly indicated?	Yes
	1.3.	Were the target population and setting specified?	Yes
2.	Was the selection of study subjects/patients free from bias?		???
	2.1.	Were inclusion/exclusion criteria specified (e.g., risk, point in disease progression, diagnostic or prognosis criteria), and with sufficient detail and without omitting criteria critical to the study?	Yes
	2.2.	Were criteria applied equally to all study groups?	Yes
	2.3.	Were health, demographics, and other characteristics of subjects described?	Yes
	2.4.	Were the subjects/patients a representative sample of the relevant population?	No
3.	Were study groups comparable?		Yes
	3.1.	Was the method of assigning subjects/patients to groups described and unbiased? (Method of randomization identified if RCT)	Yes
	3.2.	Were distribution of disease status, prognostic factors, and other factors (e.g., demographics) similar across study groups at baseline?	Yes
	3.3.	Were concurrent controls or comparisons used? (Concurrent preferred over historical control or comparison groups.)	Yes
	3.4.	If cohort study or cross-sectional study, were groups comparable on important confounding factors and/or were preexisting differences accounted for by using appropriate adjustments in statistical analysis?	N/A
	3.5.	If case control study, were potential confounding factors comparable for cases and controls? (If case series or trial with subjects serving as own control, this criterion is not applicable.)	N/A
	3.6.	If diagnostic test, was there an independent blind comparison with an appropriate reference standard (e.g., "gold standard")?	N/A
4.	Was method of handling withdrawals described?		Yes
	4.1.	Were follow-up methods described and the same for all groups?	Yes
	4.2.	Was the number, characteristics of withdrawals (i.e., dropouts, lost to follow up, attrition rate) and/or response rate (cross-sectional studies) described for each group? (Follow up goal for a strong study is 80%.)	Yes
	4.3.	Were all enrolled subjects/patients (in the original sample) accounted for?	Yes
	4.4.	Were reasons for withdrawals similar across groups?	Yes
	4.5.	If diagnostic test, was decision to perform reference test not dependent on results of test under study?	N/A
5.	Was blinding used to prevent introduction of bias?		Yes
	5.1.	In intervention study, were subjects, clinicians/practitioners, and investigators blinded to treatment group, as appropriate?	N/A
	5.2.	Were data collectors blinded for outcomes assessment? (If outcome is measured using an objective test, such as a lab value, this criterion is assumed to be met.)	Yes
	5.3.	In cohort study or cross-sectional study, were measurements of outcomes and risk factors blinded?	N/A
	5.4.	In case control study, was case definition explicit and case ascertainment not influenced by exposure status?	N/A
	5.5.	In diagnostic study, were test results blinded to patient history and other test results?	N/A
6.	Were intervention/therapeutic regimens/exposure factor or procedure and any comparison(s) described in detail? Were interveningfactors described?		???
	6.1.	In RCT or other intervention trial, were protocols described for all regimens studied?	Yes
	6.2.	In observational study, were interventions, study settings, and clinicians/provider described?	N/A
	6.3.	Was the intensity and duration of the intervention or exposure factor sufficient to produce a meaningful effect?	Yes
	6.4.	Was the amount of exposure and, if relevant, subject/patient compliance measured?	Yes
	6.5.	Were co-interventions (e.g., ancillary treatments, other therapies) described?	N/A
	6.6.	Were extra or unplanned treatments described?	N/A
	6.7.	Was the information for 6.4, 6.5, and 6.6 assessed the same way for all groups?	No
	6.8.	In diagnostic study, were details of test administration and replication sufficient?	N/A
7.	Were outcomes clearly defined and the measurements valid and reliable?		Yes
	7.1.	Were primary and secondary endpoints described and relevant to the question?	Yes
	7.2.	Were nutrition measures appropriate to question and outcomes of concern?	Yes
	7.3.	Was the period of follow-up long enough for important outcome(s) to occur?	Yes
	7.4.	Were the observations and measurements based on standard, valid, and reliable data collection instruments/tests/procedures?	Yes
	7.5.	Was the measurement of effect at an appropriate level of precision?	Yes
	7.6.	Were other factors accounted for (measured) that could affect outcomes?	Yes
	7.7.	Were the measurements conducted consistently across groups?	Yes
8.	Was the statistical analysis appropriate for the study design and type of outcome indicators?		Yes
	8.1.	Were statistical analyses adequately described and the results reported appropriately?	Yes
	8.2.	Were correct statistical tests used and assumptions of test not violated?	Yes
	8.3.	Were statistics reported with levels of significance and/or confidence intervals?	Yes
	8.4.	Was "intent to treat" analysis of outcomes done (and as appropriate, was there an analysis of outcomes for those maximally exposed or a dose-response analysis)?	N/A
	8.5.	Were adequate adjustments made for effects of confounding factors that might have affected the outcomes (e.g., multivariate analyses)?	No
	8.6.	Was clinical significance as well as statistical significance reported?	Yes
	8.7.	If negative findings, was a power calculation reported to address type 2 error?	N/A
9.	Are conclusions supported by results with biases and limitations taken into consideration?		???
	9.1.	Is there a discussion of findings?	Yes
	9.2.	Are biases and study limitations identified and discussed?	No
10.	Is bias due to study's funding or sponsorship unlikely?		???
	10.1.	Were sources of funding and investigators' affiliations described?	Yes
	10.2.	Was the study free from apparent conflict of interest?	???