Health-related quality of life and disability in young patients with spina bifida

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Health-Related Quality of Life and Disability in Young Patients With Spina Bifida Luca Padua, PhD, MD, Claudia Rendeli, MD, Alessia Rabini, MD, Elisabetta Girardi, MD, Pietro Tonali, MD, Elio Salvaggio, MD ABSTRACT. Padua L, Rendeli C, Rabini A, Girardi E, Tonali P, Salvaggio E. Health-related quality of life and disability in young patients with spina bifida. Arch Phys Med Rehabil 2002;83:1384-8. Objectives: To assess the health-related quality of life (QOL) and disability in young patients with spina bifida and to correlate them with the clinical examination findings. Design: Prospective multidimensional study by means of (1) clinical assessment, (2) self-administered questionnaire for general health, and (3) standardized disability measurements. Relationships between disability measurement, patient-oriented examination, and conventional clinical assessment were evaluated. Setting: Pediatric department at a university hospital in Italy. Patients: Twelve consecutive young patients with spina bifida (mean age, 15.2y; range, 14 –18y). Interventions: Not applicable. Main Outcome Measures: The Medical Outcomes Study 36-Item Short-Form Health Survey, the FIM™ instrument, and the Barthel Index. Results: As expected, disability was inversely related (r⫽.72, P⬍.02) to the physical aspect of QOL. Unexpectedly, for the mental aspects of QOL, less disability was associated (r⫽⫺.70, P⬍.05) with higher psychologic distress and severe role disability because of emotional problems. The findings at clinical examination, especially proximal deficit of inferior limbs (r⫽⫺.70, P⬍.05), were usually related to higher disability and lower physical aspects of QOL. Conclusion: There was no linear inverse correlation between disability and QOL in patients with spina bifida. Patients with mild disability needed as much psychologic support as patients with severe whole disability. Key Words: Disabled persons; Outcomes research; Quality of life; Rehabilitation; Spinal dysraphism. © 2002 by the American Congress of Rehabilitation Medicine and the American Academy of Physical Medicine and Rehabilitation N PATIENTS WITH SPINA BIFIDA, the major clinical problems related to the primary lesion are standing, ambuIlation, and the voluntary control of bladder and bowel function. These neurologic deficits may greatly impair the quality of life (QOL) of patients.

From the Institute of Neurology (Padua, Tonali) and Spina Bifida Center of the Paediatric Department (Rendeli, Girardi, Salvaggio), Universita` Cattolica; and Istituto Don C. Gnocchi Fondazione Pro-Iuventute (Padua, Rabini), Rome, Italy. No commercial party having a direct financial interest in the results of the research supporting this article has or will confer a benefit upon the author(s) or upon any organization with which the author(s) is/are associated. Reprint requests to Luca Padua, PhD, MD, Institute of Neurology, Universita` Cattolica, L.go F. Vito 1, 00168, Rome, Italy, e-mail: [email protected]. 0003-9993/02/8310-7278$35.00/0 doi:10.1053/apmr.2002.34599

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Health-related QOL measures are now considered to be essential,1 especially in conditions that may involve the general status of patients (eg, spina bifida).2-6 Moreover, it has been suggested that more widespread use of standardized health measures may improve clinical practice.7-12 To assess QOL in young patients with spina bifida and to correlate QOL with conventional disability measures and clinical measurements, we did a prospective multidimensional study, according to the recommended outcomes research methodology, with the most used general health status questionnaire, plus validated conventional clinical and disability measurements.5 METHODS Because self-administered questionnaires require normal cognitive function and because spina bifida is sometimes associated with brain abnormalities that may involve the intelligence quotient, we screened spina bifida patients in our pediatric department with the Wechsler Intelligence Scale for Children–Revised to exclude patients who were unable to complete the questionnaire appropriately. Of approximately 350 patients referred to the Spina Bifida Center of the Catholic University of Rome, we enrolled all who were between the ages of 14 and 18 years (42 patients). Two patients died (1 from severe Chiari malformation, 1 in a vehicular crash), 20 patients were excluded because their intelligence quotient was below 75, and 8 could not be contacted. Therefore, we enrolled 12 consecutive patients (mean age, 15.2y; range, 14 –18y). Each patient and parent was informed about the study, and they gave their consent to participate. Personal Data and Clinical Examination Before the examination was given, we acquired personal data by asking each patient to complete a case form. Questions specifically concerned the following issues: educational level (eg, school frequency, presence of assistant teacher for handicapped) and urologic aspects (incontinence, urethral catheterization, self-catheterization, urine loss between catheterizations, pharmacologic therapy). Medullar and brain magnetic resonance imaging were acquired (eg, site of lesion, type of lesion). Detailed clinical history and careful clinical examination (addressing neurologic and physical rehabilitation) were always performed (tendon reflexes of the 4 limbs, cutaneous sensitivity, muscle strength assessed according to Medical Research Council [MRC] Scale,13 joint function, trophysm, dysmetria). We summarized the clinical findings into the following measurements: muscle strength of iliopsoas and rectus femoris, MRC score of L23; muscle strength of tibialis anterior, peroneus longus, and extensor longus allucis, MRC score of L45; and muscle strength of gastrocnemius, MRC score of S1. Patient-Oriented Evaluation The Medical Outcomes 36-Item Short-Form Health Survey1,14,15 (SF-36), the most widely used generic health tool, was used in this study. The official SF-36 Italian version16 was administered to the patients according to standardized meth-

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QUALITY OF LIFE IN SPINA BIFIDA, Padua Table 1: Mean Values of SF-36 Scores and Subscores in 12 Young Patients With Spina Bifida PF

PR

BP

GH

VT

SF

RE

MH

PCS

MCS

57.6⫾29.0

68.5⫾34.1

80.3⫾23.8

63.0⫾23.1

67.9⫾15.0

67.6⫾25.4

57.5⫾26.4

72.5⫾16.0

47.3⫾12.5

48.7⫾9.0

NOTE. Values are mean ⫾ standard deviation. Abbreviations: PF, physical functioning; PR, role–physical; BP, bodily pain; GH, general health; VT, vitality; SF, social functioning; RE, role– emotional; MH, mental health.

ods.15 The SF-36 consists of 36 questions that assess the general health status of the patient. It provides 8 specific categories of physical and emotional scores (physical functioning, role–physical, bodily pain, general health, vitality, social functioning, role– emotional, mental health) summarized into 2 main scores: the Physical Composite Score (PCS) and Mental Composite Score (MCS). Very low scores on the PCS indicate severe physical dysfunction, distressful bodily pain, frequent fatigue, and unfavorable evaluation of the health status. Very low scores for the MCS indicate frequent psychologic distress and severe social and role disability because of emotional problems.15 Higher scores on the SF-36 (range, 0 –100) indicate better health. The PCS and the MCS measurements were used in the patient-oriented assessment. In some cases, we also reported the subscore results so that we could better assess the main physical and mental patterns. With regard to the subscores, we found physical role and emotional role to be very important; therefore, we specified the difference between emotional role, which assesses limitations of various kinds in everyday role activities because of emotional problems, and physical role, which assesses similar limitations because of physical health problems. Note that the SF-36 is normally used with patients more than 13 years of age.16 Disability Assessment To assess the physical disability of patients, we used 3 common measurements. The Barthel Index is considered the best scale with which to measure the daily activities. It measures performance of personal care (feeding, dressing, hygiene) and mobility (transferring, walking, wheeling); in short, it measures what an individual can do, thus providing a measure of capacity.17,18 We also used the FIM™ instrument, which is the most widely accepted functional measure.18 It consists of 18 scales, scored from 1 to 7; higher numbers mean greater ability. We used the total score.19 Finally, to evaluate walking ability, we used an adapted form (8-point scale) of the physical therapy portion of the Patient Evaluation Conference System (Deambulation Index).20

urine loss between the catheterizations; 5 of the 10 patients self-catheterized. Eight patients used a pad (7 were dependent on catheterization). Nine patients had myelomeningocele, 2 patients had lypomeningocele, and 1 had occult spina bifida. Eight patients had abnormal bowel function, ranging from constipation and urgency, to frequent and occasional accidents, and used pads or anal plugs and stomas; 4 patients had normal bowel function. Relation Between Lesion and Disability and QOL Comparison of the FIM measurement between 9 patients with lumbosacral and 3 patients with sacral lesions showed significantly (P⬍.05) lower impairment in the more caudal lesion (fig 1). Conversely, site of lesion was not correlated to the Barthel Index or Deambulation Index scores. Similarly, main QOL measurements did not correlate to the site of lesion. Relation Between QOL and Disability Measurements The physical aspects of QOL (PCS) did correlate to 2 of the disability measurements, the Barthel Index (r⫽.72, P⬍.02; fig 2A) and the Deambulation Index (r⫽.75, P⬍.02; fig 2B). Unexpectedly, the mental aspects of QOL (MCS) were inversely related (r⫽⫺.70, P⬍.05) to the Barthel Index (fig 3A): low physical disability was associated with low MCS and meant frequent psychologic distress and severe role disability because of emotional problems. The other disability measurements showed similar, although not significant, trends (fig 3B). In analyzing the subscores, we observed that the mental aspect related more to disability measurements (Barthel Index ⫺ SF-36 emotional role subscale; r⫽⫺.60, P⫽.05; Deambulation Index ⫺ SF-36 emotional role subscale: r⫽⫺.70, P⫽⫺.70, P⫽.02); note that low emotional role scores indicate a difficulty in working or daily activity due to emotional problems.15 In comparing the mental health between the 4 patients who were supported by an assistant teacher for the handicapped at

Statistical Analysis Statistical analysis was performed with the StatSoft® package.a Because an ordinal scale (eg, SF-36) was used for measurement, nonparametric analysis of the correlation was assessed by the Spearman rank-order correlation coefficient; group comparisons were assessed with the Mann-Whitney U test. RESULTS Results from the SF-36 are reported in table 1. With regard to ambulation, 8 patients were autonomous (1 with the help of a tutorial aid), while 4 required assistance. Ten patients were dependent on uretheral catheterization, 1 had continence but used a pad, and 1 had no urologic problems. Of the 10 patients who were catheter-dependent, all but 1 patient sometimes had

Fig 1. Comparison of disability between patients with lumbosacral and sacral lesions. Abbreviations: SD, standard deviation; SE, standard error.

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QUALITY OF LIFE IN SPINA BIFIDA, Padua

Relation Between Disability Measures and Clinical Measurements The FIM instrument strongly correlated to the clinical measurement of muscle function (MRC score of L23: r⫽.69, P⬍.03; MRC score of L45: r⫽.73, P⬍.02; MRC score of S1: r⫽.69, P⬍.03). Similarly, the Barthel Index score correlated to the clinical measurement of muscle function (MRC score of L23: r⫽.72, P⬍.02; MRC score of L45: r⫽.69, P⬍.03; MRC score of S1: r⫽.72, P⬍.02), as did the Deambulation Index (MRC score of L23: r⫽1.00, P⬍.00001; MRC score of L45: r⫽.65, P⬍.05; MRC score of S1: r⫽.70 P⬍.03). DISCUSSION Spina bifida is a congenital malformation of the neural tube that occurs in as many as 4.7 live births out of every 10,000.21 Myelomeningocele, the most common form of spina bifida, affects the development of the central nervous, the musculoskeletal, and the genitourinary systems. The early closure and cutaneous coverage of the myelomeningocele defect on the first day of life, and later the control of hydrocephalus with ventriculoperitoneal shunting, plus improvements in treating the neurogenic bladder, have it made possible for spina bifida patients to reach adulthood. Before 1960, as few as 10% of infants born with spina bifida survived; most died from infection and/or hydrocephalus.22 After shunt improvements and

Fig 2. Relation between disability measurements for (A) Barthel Index and (B) Deambulation Index for the physical aspects of QOL. As the level of disability increases, the physical aspect of QOL is more impaired.

school and the 8 who were not, we found a significant difference, with higher scores in the MCS (P⬍.05) and the SF-36 emotional role subscale (P⬍.03; see figs 4A, B) in the first group. Moreover, patients who were supported by an assistant teacher had higher disability: their mean FIM score ⫾ standard deviation was 91⫾20.2, while patients without an assistant teacher had a mean FIM score of 117.9⫾14 (P⬍.03). Patients with an assistant teacher had a mean Deambulation Index score of 3⫾2.8 while patients without the teacher had a mean score of 7⫾0 (P⬍.05). Patients with a teacher had a mean Barthel Index score of 57.5⫾24.7 and patients without the teacher had a mean Barthel Index score of 87.1⫾11.9 (not significant). Relation Between QOL and Clinical Picture The PCS and MCS aspects of QOL did not correlate to age, gender, or educational level. Function of lower-limb muscles, especially proximal, was strongly related to impairment of the physical aspect. In fact, the PCS correlated with an MRC score of L23 (r⫽.70, P⬍.05), an MRC score of L45 (r⫽.70, P⬍.03), and the MRC score of S1 (r⫽.78, P⬍.02). The higher the level of impairment in the lower limb, the lower the physical aspect of QOL. Arch Phys Med Rehabil Vol 83, October 2002

Fig 3. Relation between disability measurements for (A) Barthel Index and (B) Deambulation Index for the mental aspects of QOL. As the level of disability increases, the mental aspect of QOL is less impaired.

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Fig 4. Comparison of mental aspects of QOL for (A) MCS and (B) SF-36 emotional role subscale for patients with and without an assistant teacher.

more aggressive treatment, the survival rate increased. The literature23 reports a survival rate of 60% in 1963, and that rate had increased to 90% by 1974. It is estimated that from 50% to 70% of children born with spina bifida today will survive into adulthood.24 Traditional outcome assessment in neurology and rehabilitation has been based on physician-derived and instrumental findings. In the last 2 decades, clinical researchers have emphasized the need for a standardized evaluation of concepts such as QOL.25,26 Moreover, there has been recent emphasis on the need to fill a gap in the literature on disability and outcome research.11 Documenting outcomes in children and youths with chronic health conditions and disabilities is a public health priority.27 Our study assessed QOL and disability in young patients with spina bifida and correlated it with the clinical examination. In our patient sample, as expected, disability was strongly associated with clinical impairment of the proximal muscles of the lower limbs. Proximal muscles are mainly involved in walking and in the motor function of the lower limbs. This relationship shows that clinical examination is useful in assessing the severity of the disease because it provides a measure of the muscle deficit and because it appears to be related to the patient’s disability. With regard to QOL, the pattern appears more complex. Expectedly, greater disability and severe muscle deficit of the

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lower limbs were associated with a reduction in the physical aspect of QOL. However, unexpectedly, the mental aspects of QOL had the opposite relation to disability: patients with low disability, for example, normal walking ability or total autonomy, had more emotional problems (MCS) than patients with severe disability. When we analyzed which emotive aspect was mainly involved by evaluating the subscores, we found that the patients with less disability had difficulty in daily activity because of emotional problems. Conversely, patients with high disability (inability to walk, sphincter problems, total need for assistance) indicated that their inability to do daily activities was a result of physical problems and that, from an emotional point of view, they had a good QOL. Our data confirm the previous observation by Minchom et al,28 who found that greater feelings of global self-worth and self-esteem in physical appearance were associated with greater severity of disability. We hypothesized that the sphincter problem plays a key role in this QOL pattern; in fact, it must be noted that even patients with low disability have severe sphincter problems (all but 2 of our subjects were catheterized or used a pad). Very likely, patients with lower disability have more social activity and spend most of their time among healthy people, but they conceal their severe urologic problems. Conversely, patients with severe disability perceive that their limitations are not because of emotional problems but are a result of physical problems. Therefore, because of the evidence of their disability, they accept their condition emotionally. Moreover, we observed that the presence of a teaching assistant at school was associated with a better mental QOL. We suggest 2 reasons for this association: (1) all Italian schools provide a teaching assistant for patients who have more severe physical problems (this is confirmed by the significant correlation between disability scores and the presence of an assistant), and (2) the assistant teacher may have a positive impact on emotional QOL. With regard to the emotional health, we believe that parents greatly influence this aspect of patient QOL. Some studies assess parents’ perception of QOL and the perceptions of youths, but the relationship is complex and still a matter of debate.29,30 A study that is focused on this issue is underway. The main shortcoming of this study was its small sample size. Our department plays a key role in the diagnosis and treatment of a large population of spina bifida patients, but we studied only a small sample because of the strict criteria used and the specific aim of the study. Notwithstanding, this comprehensive and multiperspective assessment of the disease yielded significant results. CONCLUSION Our results provide data that disputes an easy and linear inverse correlation between disability and QOL in patients with spina bifida. Assessment of QOL, and particularly the emotional results, gives us useful information for clinical practice: patients with mild disability who are able to walk, run, and have total autonomy, but who have urologic problems, need psychologic support more than do patients with severe disability. Moreover, while these results should provoke debate on how these patients should be supported, only focused studies will provide definitive information. Acknowledgment: We thank Pietro Caliandro and Cristina del Prete for technical support. Arch Phys Med Rehabil Vol 83, October 2002

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