The Impact of a Slow-Release Large Neutral Amino Acids Supplement on Treatment Adherence in Adult Patients with Phenylketonuria
<p>Patients’ subjective assessment of the level of therapy adherence over the past 4 weeks.</p> "> Figure 2
<p>Frequency of non-adherence expressed by 3 questions relating to frequency over the past 4 weeks of (<b>A</b>) "Leftover drug," (<b>B</b>) "Insufficient stock of drug," and (<b>C</b>) "Missed administration of drug."</p> ">
Abstract
:1. Introduction
2. Materials and Methods
2.1. Study Population
2.2. Methods for Therapy Adherence Assessment
2.3. Methods for Quality of Life Assessment
2.4. Statistical Analysis
3. Results
3.1. Biochemical Measurement
3.2. Medication Adherence Pre- and Post-LNAA
3.3. PKU-QoL Pre- and Post-LNAA Introduction
4. Discussion
5. Conclusions
Supplementary Materials
Author Contributions
Funding
Acknowledgments
Conflicts of Interest
References
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Patients | Gender | Age, Years | Molecular Analysis | Marital Status | Educational Level 1 | Professional Status | Leisure Time |
---|---|---|---|---|---|---|---|
1 | M | 38 | c.473G>A/ c.1315+1G>A | Unmarried | 8 | Agriculture | Not specified |
2 | M | 33 | c.473G>A/ c.526C>T | Married | 18 | Insurance agent | Sport |
3 | M | 30 | c.473G>A/ c.473G>A | Married | 18 | Unemployed | Intellectual |
4 | M | 35 | c.842+3G>C in heterozygosis | Married | 13 | Office worker | Not specified |
5 | M | 26 | c.47_48delCT/ c.1315+2T>C | Unmarried | 18 | Freelance | Intellectual |
6 | F | 32 | c.842C>T/ c.1315+1G>A | Married | 18 | Teacher | Social activity |
7 | F | 28 | c.842C>T/ c.1315+1G>A | Unmarried | 13 | Office worker | Not specify |
8 | F | 22 | c.842C>T/ c.1315+1G>A | Unmarried | 13 | Unemployed | Social activity |
9 | M | 32 | c.1222C>T/ c.1315+1G>A | Unmarried | 13 | Sales | Not specify |
10 | M | 40 | c.782G>A/ c.782G>A | Married | 13 | Freelance | Sport |
11 | F | 20 | c.782G>A/ c.1066-11G>A | Unmarried | 13 | Unemployed | Not specify |
12 | F | 19 | c.1222C>T/ macro deletion in exon 3 | Married | 13 | Unemployed | Sport |
Patient | 12 Months before LNAA | 12 Months after LNAA | ||||||
---|---|---|---|---|---|---|---|---|
Phe Values | Tyr Values | Phe/Tyr Values | DBS Frequency | Phe Values | Tyr Values | Phe/Tyr Values | DBS Frequency | |
1 | 790 ± 80 | 47 ± 8 | 17.2 ± 2.5 | 8 | 825 ± 114 | 68 ± 10 | 12.4 ± 2.4 | 17 |
2 | 1033 ± 198 | 54 ± 10 | 19.5 ± 4.4 | 5 | 1269 ± 265 | 65 ± 14 | 20.6 ± 7.6 | 14 |
3 | 983 ± 142 | 32 ± 7 | 31.5 ± 7.3 | 10 | 907 ± 166 | 60 ± 8 | 15.4 ± 3 | 20 |
4 | 790 ± 118 | 83 ± 14 | 9.7 ± 2.2 | 18 | 769 ± 144 | 98 ± 18 | 8 ± 1.4 | 34 |
5 | 838 ± 179 | 57 ± 12 | 15.7 ± 6.6 | 20 | 889 ± 149 | 79 ± 19 | 11.7 ± 2.7 | 24 |
6 | 880 ± 160 | 49 ± 11 | 18.3 ± 4 | 38 | 975 ± 148 | 68 ± 29 | 16 ± 5 | 39 |
7 | 779 ± 108 | 87 ± 35 | 10.6 ± 5 | 25 | 736 ± 93 | 86 ± 41 | 10.5 ± 4.9 | 27 |
8 | 628 ± 148 | 50 ± 13 | 13 ± 5.2 | 26 | 790 ± 147 | 59 ± 15 | 14 ± 4.5 | 29 |
9 | 808 ± 135 | 59 ± 7 | 19.9 ± 2.8 | 12 | 925 ± 139 | 79 ± 13 | 11.9 ± 1.8 | 18 |
10 | 842 ± 94 | 75 ± 12 | 11.5 ± 2.3 | 23 | 879 ± 91 | 108 ± 35 | 8.5 ± 1.9 | 36 |
11 | 765 ± 234 | 58 ± 11 | 13.4 ± 3.9 | 23 | 760 ± 124 | 60 ± 11 | 13 ± 2.7 | 24 |
12 | 823 ± 117 | 53 ± 19 | 16.9 ± 5 | 18 | 1000 ± 163 | 68 ± 17 | 15.2 ± 3.7 | 15 |
Overall Patient Population | Prior to LNAA Treatment | During LNAA Treatment | p Value |
---|---|---|---|
Phe | 752 ± 143 | 894 ± 145 | 0.0522 |
Tyr | 59 ± 13 | 75 ± 16 | 0.0195 |
Phe/Tyr | 16± 4 | 12 ± 3 | 0.049 |
Dimension | Item | p Value |
---|---|---|
Your Health | In the past 7 days, I became aggressive | 0.044 |
Your PKU diet and supplements | In the past 7 days, it was hard to take my supplements several times a day | 0.042 |
In the past 7 days, it was hard to only eat/drink what I should | 0.047 | |
In the past 7 days, my supplements tasted: very good/good/ok, bad, very bad/I don’t take supplements | 0.017 | |
In the past 7 days, I thought about eating food that was not on my diet | 0.047 | |
Your daily life with PKU | Currently, it is inconvenient to carry my supplements with me when I am going on business trips or on holiday | 0.040 |
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Burlina, A.P.; Cazzorla, C.; Massa, P.; Loro, C.; Gueraldi, D.; Burlina, A.B. The Impact of a Slow-Release Large Neutral Amino Acids Supplement on Treatment Adherence in Adult Patients with Phenylketonuria. Nutrients 2020, 12, 2078. https://doi.org/10.3390/nu12072078
Burlina AP, Cazzorla C, Massa P, Loro C, Gueraldi D, Burlina AB. The Impact of a Slow-Release Large Neutral Amino Acids Supplement on Treatment Adherence in Adult Patients with Phenylketonuria. Nutrients. 2020; 12(7):2078. https://doi.org/10.3390/nu12072078
Chicago/Turabian StyleBurlina, Alessandro P., Chiara Cazzorla, Pamela Massa, Christian Loro, Daniela Gueraldi, and Alberto B. Burlina. 2020. "The Impact of a Slow-Release Large Neutral Amino Acids Supplement on Treatment Adherence in Adult Patients with Phenylketonuria" Nutrients 12, no. 7: 2078. https://doi.org/10.3390/nu12072078