Assessment of Quality of Life on Home Parenteral Nutrition and After Intestinal Transplantation Using Treatment-Specific Questionnaires
In order to investigate the quality of life on home parenteral nutrition and after intestinal transplantation using comparable questionnaires, the treatment-specific quality of life questionnaire for adult patients on home parenteral nutrition was adapted for intestinal transplant recipients. Both instruments were composed of 8 functional scales, 9 symptom scales, 3 global health status/quality of life scales and 2 single items. A preliminary cross-sectional study enrolling all the patients currently cared at the same hospital was carried out. Exclusion criteria were age ≥ 60 years and hospitalization at time of assessment. Thirty-three home parenteral nutrition patients (100% answered) and 22 intestinal transplant recipients (82% answered) were enrolled. Intestinal transplant recipients showed a better score in following scales: ability to holiday/travel (p < 0.001), fatigue (p = 0.022), gastrointestinal symptoms (p < 0.001), stoma management/bowel movements (p = 0.001) and global health status/quality of life (p = 0.012). A better score for ability to eat/drink (p = 0.070) and a worse score for sleep pattern (p = 0.100) after intestinal transplantation were also observed. The results of this preliminary study with specific instruments were consistent with the main expected improvement of the quality of life related to intestinal transplantation. Further studies in larger patient cohorts are required to confirm these data.
quality of life
home parenteral nutrition
Quality of Life Inventory
Home parenteral nutrition (HPN) is the primary treatment for irreversible intestinal failure (IF) and intestinal transplantation (ITx) is the life-saving treatment for those patients who are at risk of death because of life-threatening complications of HPN or of underlying nonmalignant gastrointestinal diseases (1). The results of ITx have improved over the last decade (2). When long-term survival rate after ITx, as well as the rate of treatment-associated death, become similar to those on HPN, ITx could be proposed as a rehabilitative procedure to all the patients with irreversible IF (3,4).
Rehabilitation is not only related to the recovery of intestinal autonomy but also to an improvement in the quality of life (QoL). In the context of health, QoL describes health status from the patients’ perspective (5–7). Studies have shown that adult patients receiving HPN for benign IF have a poorer QoL than that of the normal population and of some patients with chronic illness (5,6). The few studies that compared the health-related QoL of patients on HPN with that of adult ITx recipients showed a modest improvement of QoL posttransplant (7–11). In these studies, the instruments used to assess QoL were generic health status questionnaires (10,11) and a treatment-specific tool designed for transplant recipients (8,9). None of the used instruments were specifically designed for either HPN patients or ITx recipients.
Recently, a specific tool for the assessment of QoL in adult patient on HPN for benign IF has been validated (HPN-QOL©) (12). In order to compare the QoL on HPN and after ITx using comparable specific instruments, the HPN-QOL© was adapted to ITx recipients, and a cross-sectional study was carried out investigating HPN patients and ITx recipients who were cared for at the same hospital.
Materials and Methods
Adaptation of the HPN-QOL© instrument to ITx
The HPN-QOL© is a 48-item questionnaire that focuses on physical, emotional and symptomatic issues (12). The questionnaire contains 7 multi-item functional scales and 1 single-item functional scale, as well as 6 multi-item and 3 single-item symptom scales. The functional scales include general health, ability to holiday (vacation) or travel, coping, physical function, ability to eat and drink, employment, sexual function and emotional function. The symptom or problem scales include body image, immobility, fatigue, sleep pattern, gastrointestinal symptoms, other pain, either stoma management or bowel movements, financial issues and body weight. There are three global health status/quality of life numerical rating scales. The first is a global QoL question. The other two numerical rating scales pertain to the effect on QoL of the underlying illness leading to the need for HPN and the effect of the HPN. Two single HPN items relate to nutrition team and the availability of an ambulatory pump for infusion of HPN, in which a high score represents a good outcome.
The adaptation of the HPN-QOL© instrument to ITx recipients was done by modifying the text of some items, without changing the number of either the items or the scales. Only those items that contained the term HPN or that were linked to the HPN were changed (see Supplementary Material).
This was a cross-sectional study. All the patients currently followed at the Center for Chronic Intestinal Failure and at the Transplantation Unit of the S. Orsola-Malpighi University Hospital of Bologna, Italy, who were on HPN for benign IF or who had undergone ITx were invited to participate in the study. Invitation was done by a physician (LP) in person or by phone. Exclusion criteria were: age ≥ 60 years and hospitalization at time of invitation.
The questionnaire was filled in by the patients at time of a scheduled visit at the outpatient clinic or was mailed to the patient at home, after the informed consent was obtained. Informed consent was obtained in person or by phone at time of invitation, before administering or mailing the questionnaire. Verbal instructions about filling in the questionnaire were given at time of obtaining the informed consent. When required, assistance in filling in was given in person or by phone. The questionnaire was mailed twice to those patients who did not respond to the first mailing.
At the same time as completing the questionnaire, the following sociodemographic, general and underlying disease-related factors were recorded for each patient: age, sex, underlying disease, cause of intestinal failure, primary reason for HPN, duration of HPN or of ITx, functional status, living status, marital status, educational status, employment status, presence of a stoma; for patients on HPN: HPN supplemental or total, number of HPN infusion per week, duration of infusion (hours), home care company support, being on waiting list for ITx; for ITx recipients: indication for ITx, type of ITx, intestinal functional autonomy.
The following data about the intensity of medical care required by the patients were collected with respect to the date of the questionnaire administration: number of clinic visits, number of hopitalizations and of hospital days during the year preceding the questionnaire administration; days from the last hospital discharge.
The study was approved by the institution review board and ethical committee: approval number 1248/2007.
The HPN-QOL© scoring system has been previously reported (12). All scales are linearly transformed to scores between 0 and 100. For the functional and health status/quality of life scales and the two HPN items, a high score indicates high functioning or good health status; for the symptom scales/items a high score represents a high (“bad”) level of symptoms.
Variables are reported as mean ± standard deviation or percentages. Differences between means were analyzed by the Student t-test. Differences between frequencies were analyzed by the χ2 test. The Statgraphics® Centurion XV statistical package 2008 (StatPoint, Inc., Warrenton, VA, USA) was used for the analyses.
P values of < 0.05 were considered significant. P values ≥ 0.05 and ≤ 0.10 were described as a nonsignificant trend.
When a statistically significant difference between the HPN and the ITx groups was observed for the means of a given scale of the QoL questionnaires, the magnitude of the difference was estimated by the measurement of the effect size, using the Cohen's d index (13). According to the d value, the effect size is defined trivial (d < 0.20), small (d = 0.20 to 0.49), medium (d = 0.50 to 0.79), large (d > 0.80).
Thirty-three HPN patients and 22 ITx recipients were invited to participate in the study. All the HPN-patients and 18 ITx recipients filled in the questionnaire. Two ITx recipients who did not participate were too sick, one was wheelchair bound because of neurological problems and one had major depression. Two ITx recipients, known to be in good clinical condition, did not respond to the invitation. All the four ITx recipients who did not respond had been invited by mail.
The characteristics of the two patient cohorts who participated in the study are shown in Table 1. Statistically significant differences were found for: gender (more female in HPN); hospitalization and clinic visit rates (greater in HPN), underlying gastrointestinal disease (more Crohn's disease and chronic idiopathic intestinal pseudo-obstruction in HPN); duration of the treatment (more HPN patients with duration < 2 years) and employment status (more unemployed in HPN). A near significant difference was observed for age (younger in ITx) and marital status (more single in ITx).
Table 1. Characteristics of the patient cohorts who filled in the questionnaire. Categorical data are reported as number (% of cohort population)
|M||12 (39)||12 (67)||0.038|
|F||21 (61)||6 (33)|| |
|Age (years)||44 ± 10||39 ± 10||0.083|
|Mode of questionnaire administration (filling in)|| || ||0.213|
| In person (at clinic visit)||24 (73)||10 (56)|| |
| By mail (at home)||9 (27)||8 (44)|| |
|Patients who were hospitalized during the year preceding the questionnaire administration||21 (64)||3 (17)||0.002|
| Hospitalizations per patient||2.33 ± 1.27||1.00 ± 0||0.009|
| Days of hospitalization per patient||27 ± 26||50 ± 28||0.179|
| Days between the last hospitalization and questionnaire administration||107 ± 82||131 ± 57||0.641|
|Hospitalization characteristics in patients who were hospitalized|| || || |
| Hospitalizations per patient||2.33 ± 1.27||1.00 ± 0||0.009|
| Days of hospitalization per patient||27 ± 26||50 ± 28||0.179|
| Days between hospitalization and questionnaire administration||107 ± 82||131 ± 57||0.641|
|Clinic visits per patient during the year preceding the questionnaire administration||4.66 ± 2.65||2.83 ± 1.54||0.009|
|On waiting list for ITx||5 (15)|| || |
| Indication for ITx|| || || |
| Liver failure (impending)||1|| || |
| Intraabdominal desmoids||1|| || |
| Ultra short bowel||2|| || |
| Frequent hospitalization/HPN refusal||1|| || |
|Underlying disease|| || ||0.047|
| Crohn's disease||9 (27)||0|| |
| Mesenteric ischemia||4 (12)||7 (39)|| |
| Chronic idiopatic pseudo-obstruction||10 (30)||4 (22)|| |
| Intestinal volvulus||7 (21)||4 (22)|| |
| Others||3 (9)||3 (17)|| |
|Cause of intestinal failure|| || ||0.409|
| SBS||18 (55)||13 (72)|| |
| Motility disorder||10 (30)||4 (22)|| |
| Others||5 (15)||1 (6)|| |
|Duration of ongoing HPN or of ITx|| || ||0.008|
| ≤ 2 years||20 (61)||4 (22)|| |
| > 2 years||13 (39)||14 (78)|| |
|Functional status|| || ||0.619|
| Independent||24 (73)||12 (67)|| |
| Require some help||8 (24)||6 (33)|| |
| Require total help||1 (3)||0|| |
|Living status|| || ||0.341|
| Alone||3 (9)||4 (22)|| |
| Family||29 (89)||14 (78)|| |
| With other adults||1 (3)||0|| |
|Marital status|| || ||0.077|
| Single||8 (24)||10 (56)|| |
| Married||20 (60)||6 (33)|| |
| Separated||1 (3)||2 (11)|| |
| Divorced||3 (9)||0|| |
| Widow (er)||1 (3)||0|| |
|Educational status|| || ||0.337|
| Less than compulsory school education||3 (9)||0|| |
| Compulsory school education||11 (33)||6 (33)|| |
| Post compulsory school, below university||14 (43)||11 (61)|| |
| University level||5 (15)||1 (6)|| |
|Employment status|| || ||0.013|
| Employed full time||4 (12)||7 (39)|| |
| Employed part time||6 (18)||3 (17)|| |
| Homemaker||2 (6)||1 (6)|| |
| Student||0||1 (6)|| |
| Unemployed||17 (52)||1 (6)|| |
| Retired||4 (12)||5 (27)|| |
|Presence of a stoma|| || ||0.534|
| No||21 (64)||13 (73)|| |
| Ileostomy||12 (36)||5 (27)|| |
|HPN characteristics|| || || |
| Total||8 (24)|| || |
| Supplemental||25 (76)|| || |
| Infusions per week|| || || |
| 2–3||7 (21)|| || |
| 4–5||5 (15)|| || |
| 6–7||21 (64)|| || |
| Duration of infusion|| || || |
| 9–13 h||16 (49)|| || |
| 14–18 h||13 (39)|| || |
| 19–24 h||4 (12)|| || |
| Home care company support|| || || |
| Yes||22 (67)|| || |
| No||11 (33)|| || |
|ITx characteristics|| || || |
| Indication for ITx|| || || |
| Liver failure|| ||2 (11)|| |
| Multiple Vein thrombosis|| ||2 (11)|| |
| Intraabdominal desmoids|| ||2 (11)|| |
| Ultra short bowel|| ||8 (44)|| |
| Frequent hospitalization/HPN refusal|| ||4 (22)|| |
| Type of ITx (n.)|| || || |
| Isolated small bowel|| ||15 (83)|| |
| Multivisceral without liver|| ||1 (6)|| |
| Multivisceral with liver|| ||2 (11)|| |
| Intestinal functional autonomy|| || || |
| Full|| ||15 (83)|| |
| Need IV hydration|| ||1 (6)|| |
| Need HPN|| ||2 (11)|| |
The results of the HPN-QOL© in the total groups of patients are reported in Table 2. ITx recipients showed a statistically significant better score in ability to holiday/travel, fatigue, gastrointestinal symptoms, either stoma management or bowel movements and the numerical rating scales of quality of life. ITx recipients also showed a trend for a better score for ability to eat/drink (p = 0.070) and a worse score for sleep pattern (p = 0.100).
Table 2. Results of the HPN-QOL© and its adapted version for ITx in the two cohorts of patients
|Functional scales (high score = good functioning)|
| General health||33||78.0 ± 24.8||18||80.5 ± 16.1||−0.38||0.699|
| Ability to holiday/travel||33||25.7 ± 16.2||18||48.6 ± 23.4||−4.09||0.000|
| Physical function||31||48.1 ± 13.3||18||43.9 ± 20.9||0.84||0.402|
| Coping||30||58.1 ± 18.3||16||50.0 ± 18.7||1.42||0.161|
| Ability to eat /drink||32||56.2 ± 23.4||18||68.5 ± 20.5||−1.85||0.070|
| Employment||29||40.2 ± 27.2||18||50.0 ± 29.1||−1.16||0.251|
| Sexual function||26||23.0 ± 29.0||13||32.0 ± 25.8||−0.94||0.352|
| Emotional function||32||65.2 ± 23.1||17||69.1 ± 26.3||−0.52||0.600|
| Nutrition/hospital team support||33||79.7 ± 32.2||18||77.7 ± 22.8||0.23||0.814|
|Symptom scales (high score = worse/symptom)|
| Body image||33||27.2 ± 25.6||18||27.7 ± 30.7||−0.06||0.950|
| Body weight||33||26.2 ± 33.0||18||35.1 ± 40.3||−0.85||0.398|
| Immobility||33||23.8 ± 17.5||18||23.3 ± 21.8||0.08||0.928|
| Fatigue||33||37.3 ± 26.0||18||20.3 ± 21.8||2.35||0.022|
| Sleep pattern||33||18.1 ± 31.2||18||35.1 ± 40.3||−1.67||0.100|
| Gastrointestinal symptoms||32||36.8 ± 17.0||18||12.3 ± 13.6||5.21||0.000|
| Other pain||33||28.2 ± 24.4||18||19.4 ± 19.1||1.32||0.191|
| Stoma management or bowel movements||30||35.0 ± 27.2||16||10.7 ± 9.3||3.43||0.001|
| Financial issues||26||20.5 ± 31.3||17||19.6 ± 29.0||0.09||0.924|
| Numerical rating scales of quality of life (high score = good Qol)||30||35.7 ± 18.9||17||52.3 ± 24.3||−2.60||0.012|
Taking into consideration the statistically significant differences in the characteristics of the two cohorts, the analysis was repeated in the following subgroups of patients: male patients (HPN 12; ITx 12); patients who had no hospitalization during the preceding year (HPN 12, ITx 15); HPN patients who were not listed for ITx (HPN 28, ITx 18); SBS as cause of IF (HPN 18, ITx 13); mesenteric ischemia or volvulus as underlying disease (HPN 11, ITx 11); duration of the treatment > 2 years (HPN 13, ITx 14); single, separated, divorced or widow/er (HPN 13, ITx 12); employed full or part time (HPN 10, ITx 10). In all the subgroups, the results confirmed those observed in the whole cohorts, with the exception of the employed subgroup, where HPN patients had also a worst score for employment (HPN, 46 ± 23; ITx 70 ± 13; p = 0.012) and for emotional function (HPN, 55 ± 21; ITx 79 ± 24; p = 0.031).
A comparison was performed between patients who filled in the questionnaire during a clinic visit and those who filled in at home. Both the clinical and sociodemographic characteristics and the HPN-QOL© scales were analyzed. Within the group of ITx recipients, no statistically significant difference was observed. Within the HPN group, patients who filled in the questionnaire at a clinic visit showed a longer time (days) between hospitalization and questionnaire administration (128 ± 79 vs. 28 ± 23; p = 0.025), a lower rate of independent functional status (62% vs. 100%; p = 0.031), and a better score in the ability to holiday/travel scale (31 ± 14 vs. 11 ± 11; p < 0.001) and in the fatigue scale (31 ± 23 vs. 51 ± 29; p = 0.048).
The comparison between the HPN and the ITx subgroups of those who filled in the questionnaire at the clinic visit and of those who filled in at home, were similar to the results observed in the whole cohorts (Table 2).
The estimation of the effect size was performed in both the whole group and the subgroups (Table 3). The magnitude of the observed statistical significant differences was always large for the ability to travel, the gastrointestinal symptoms and the stoma management/bowel movements scales, whereas it ranged from small to large for the fatigue scale and from medium to large for the numerical rating scale of quality of life.
Table 3. Effect size analysis of the observed statistical significant differences calculated by the Cohen's d index. Magnitude of the observed differences: trivial (d < 0.20), small (d = 0.20 to 0.49), medium (d = 0.50 to 0.79), large (d > 0.80)
|Ability to holiday/travel||1.13||0.98||1.23||1.08||1.18||1.33||1.02||1.01||1.28||1.01||1.73|
|Stoma management or bowel movements||1.19||0.58||1.49||1.15||1.12||0,87||1.14||1.13||1.70||1.36||0.86|
|Numerical rating scales of quality of life||0.76||0.87||1.05||0.76||0.68||0.68||0.67||1.27||1.36||0.55||1.15|
The results of this preliminary cross-sectional study indicate that ITx could improve the patient QoL with respect to HPN, due to a reduction in gastrointestinal symptoms and fatigue, enjoyment in eating and freedom of moving without the burden of HPN facilities (5–7,14). The observed improvements were probably the main reasons for the higher score in the global health status/QoL numerical rating scale observed in ITx recipients, as no differences or trends were observed in the other functional or symptoms scales of the questionnaires. The analysis of the subgroups of patients confirmed the results observed in the whole cohorts, with the exception of the subgroup of those who were employed, where ITx recipients showed a better score in the scales of ability to go to work and of emotional function. This would suggest that a successful ITx was also associated with less uncertainness concerning the employment status and thus with less anxiety and/or depression.
Although the observed results were consistent with the main expected improvement of QoL related to ITx, they cannot be considered conclusive. Indeed, the analysis of the effect size showed that the magnitude of the observed differences was large for the ability to travel, the gastrointestinal symptoms and the stoma/bowel management scales in all the patient groups, whereas for the fatigue and the global health status/QoL numerical rating scales it ranged from small to large, indicating the need for investigations on larger patient cohorts to support these findings. The differences in the clinical and sociodemographic characteristics as well as in the responder rates between the two cohorts, and the lack of validation of the adapted version of the HPN-QOL© version adapted for ITx recipients are the major limitations of the study. The small sample populations did not allow us to define the factors that could influence the QoL of both HPN and ITx patients. The 20% missing questionnaires in ITx recipients compared to the 100% responses in the HPN group could be a bias because two out of the four ITx patients who did not answer the questionnaire were known to have a good clinical status, but two were in a poor clinical condition. The clinical and psychometric analysis of the HPN-QOL© version adapted for ITx recipients will allow its validation (12). This was beyond the aim of this pilot study.
There are few previous studies about QoL after ITx in adults to be compared with the present one. Cameron et al. using the generic instrument Short Form 36 (SF-36) to compare four patients after ITx with 12 stable patients on HPN failed to reveal differences between the groups (10). Similarly, using the SF 36 questionnaire we compared 18 patients stable on HPN and 12 patients who underwent successful ITx, observing that only the body pain score differed significantly between the groups, with a better value in ITx recipients (11). The only studies using a disease-specific instrument were carried out a decade ago in Pittsburgh (8,9). The Quality of Life Inventory (QOLY), designed for transplant recipients, validated in liver transplant patients, but not for HPN patients, was used. Di Martini et al. performed a retrospective study on nine ITx recipients, who rated QOLY for three time periods: premorbid (pre-HPN), morbid (HPN) and after small bowel transplant (post-ITx) (8). When comparing pre-HPN to HPN, a disruption in most areas of their lives was reported, except for marital relationships, medical compliance and medical satisfaction. When comparing post-ITx status to that during HPN, ITx recipients experienced a significant improvement in most areas of QoL, psychological, physical and social. For nine domains alcohol use, drug use, cognitive and emotional style, pain and discomfort, finances, marital relationships, medical compliance, medical satisfaction and parenting-QoL did not show significant changes. Rovera et al. published a cross-sectional analysis that compared QOLY of 10 patients on HPN with that of 10 ITx recipients (9). ITx recipients showed a worst score for drug use and medical compliance. ITx patients reported requiring more medication for sleep, which accounted for their increased drug use. No other statistically significant difference was found between the two groups; even digestive and urinary symptoms were rated with the same score. It is difficult to compare the results obtained with QOLY with those of this study obtained with HPN-QOL© and its version for ITx recipients. However, it seems that in both cases, no difference was observed between HPN patients and ITx recipients in most of the scales or domains of QoL. Furthermore, the increased need for medication for sleep, observed by Rovera in ITx recipients, fits with the worse numerical score of sleep pattern in ITx recipients observed in our study. On the other hand, HPN-QOL© and its version for ITx recipients were able to clearly report the impact of both treatments on the gastrointestinal symptoms, eating and drinking and patient moving around.
The results of our study would support the rehabilitative role of ITx for patients with irreversible IF, but the available data do not yet allow us to consider poor QoL on HPN a straight away indication for ITx. An ITx for a low QoL on HPN could be offered when the survival probability after ITx will be definitely similar or better than that on HPN. The results of ITx have improved over the past decade. Single-center series report 1- and 5-year patient and graft survival to be 78–85% and 56–61%, respectively (2,7). However, these rates seem still lower than the 84% 5-year survival rate observed in stable patients on HPN having no factors associated with an increased risk of death (4). We also need to define the QoL threshold on HPN for offering a rehabilitative ITx. Many factors associated with poor QoL on HPN have been described (5): sudden onset of the intestinal failure in previously fit and well individuals who have suffered a catastrophic event such as mesenteric thrombosis or volvulus, not eating or drinking normally, loss of independence with limitations in social life and lack of freedom on HPN dependence, change in social status as a result of not being able to go out to work and the implications this may have for the patient financial status, worsening nutritional status, increased number of HPN infusions per week, frequency of nocturnal urination, need of opiates to control pain. However, all but one the published studies had a cross-sectional design (5). The only one prospective study was a 1-year follow-up performed on a small patient population, using the generic tool SF 36 (15). The implementation of the HPN-QOL© in the clinical practice will allow a better definition of factors affecting QoL on HPN and the identification of the threshold for referral for ITx.
In conclusion, the results of this preliminary study, obtained using the HPN-QOL© and its adapted version for ITx recipients, as treatment-specific instruments to evaluate QoL in patients with irreversible IF, were consistent with the main expected improvement of the QoL related to ITx. The validation of the HPN-QOL© adapted for ITx and studies in larger patient cohorts are required to confirm and further develop these data.
The authors of this manuscript have no conflicts of interest to disclose as described by the American Journal of Transplantation.