Quality of life assessment in adults with type 1 Gaucher disease.

The effect of enzyme replacement therapy on health-related quality of life in 25 adults with type 1 Gaucher disease was investigated over a 2-year period. Quality of life was assessed using the SF-36 Health Survey (SF-36). Psychological functioning was assessed using the Symptom Checklist--90R. The results indicated significant improvement in 7 of 8 SF scale scores beginning at 18 months of therapy (P < 0.05 to 0.001). The SF scale showing improvement first was Vitality (energy level and fatigue) at 6 months of therapy (P < 0.01). The SF-36 scales showing the largest improvements were Role-Physical and Social Functioning (P < 0.001). Compared to the general US adult population, the study population's health profile was significantly lower prior to starting therapy but by 24 months of therapy there were no differences between the two. No differences were found in psychological functioning compared to a US adult normative group at the start of therapy. However, within the study population there was significant improvement in mood and global functioning and fewer psychological symptoms reported at 24 months of therapy. The findings indicate that enzyme replacement therapy for type 1 Gaucher disease has a positive impact on health-related quality of life from the patient's perspective.

Quality-of-life assessment after ileoanal pull-through for ulcerative colitis and familial adenomatous polyposis.

BACKGROUND/PURPOSE: The ileoanal pull-through procedure (IAP) is gaining increasing favor and use in the surgical treatment of children with ulcerative colitis (UC) and familial adenomatous polyposis (FP). Although physiological studies have been performed to assess the outcome of these children, no long-term quality-of-life assessment after the procedure has been performed. METHODS: Forty-three patients were identified who had an IAP at our institution in the last 10 years and were at least 6 months postsurgery. Thirty-four were contacted, and 32 agreed to participate in the survey, which was approved by the Human Studies Committee. Participants completed the standardized Medical Outcome Study Short Form-36 (SF-36), which has well-established normative values. Several supplemental questions were prepared in a similar format dealing with issues specific to the ileoanal pull-through procedure. RESULTS: Of the 32 participants, 19 (59%) were girls and 26 (81%) had ulcerative colitis. Mean age at the time of survey was 18.1 years with 12 less than 18 years and 20 > or =18 years. Data from the latter group could be compared with national normative values for this age. The study group was not statistically different from age-appropriate US population normal values on all assessable scales of physical and mental health in the SF-36 survey including physical functioning, role limitations-physical, bodily pain, general health, vitality, social functioning, role limitations-emotional, and mental health (all P>.05 or mean difference SD units <0.8). The supplemental questionaire demonstrated little adverse effect of the surgery. There was limited consumption of medications to control bowel frequency and little restriction of activity because of the frequency of bowel movements or fear of incontinence. The surgical scar was the sole negative factor of significance. CONCLUSIONS: The ileoanal pull-through procedure is an excellent surgical option for children with ulcerative colitis or familial adenomatous polyposis, and it produced minimal, if any, adverse effects on their long-term quality of life.

Reflex sympathetic dystrophy in children. Clinical characteristics and follow-up of seventy patients.

We report on the experience with our first seventy patients who had reflex sympathetic dystrophy and were less than eighteen years old (average age, 12.5 years). In our series, the patients were predominantly girls (male to female ratio, 11:59) and the lower extremity was involved most often (sixty-one of the seventy patients). The average time from the initial injury to the diagnosis was one year, which indicates that the syndrome remains under-recognized in patients in this age-group. Conservative treatment with physical therapy, transcutaneous electrical nerve stimulation, psychological therapies including cognitive-behavioral management and relaxation training, and tricyclic anti-depressants was effective in improving the average scores for pain and function for forty patients. Sympathetic blocks were helpful for twenty-eight of thirty-seven patients. Thirty-eight of the seventy patients in the series continued to have some degree of residual pain and dysfunction. Reflex sympathetic dystrophy in children differs in presentation and clinical course from the syndrome in adults. It is best treated in a multidisciplinary fashion.

Depression in a pediatric intensive care unit nursing staff.

This study compared the depressive symptoms of 36 ICU nurses to 23 non-ICU nurses. The study was conducted in a pediatric setting where critically ill children may present unique demands. The ICU nursing staff had a significantly higher frequency (28%) of symptoms consistent with a depressive disorder than either the age group norm (12%) or the non-ICU nurses (13%). This finding suggests a continued need for investigation into the stresses associated with the delivery of critical care.

Temporomandibular joint dysfunction and facial pain in children: an approach to diagnosis and treatment.

A model for the diagnosis and treatment of temporomandibular joint dysfunction and facial pain in children is presented. Emphasis is placed on systematic assessment of physical, psychologic, and behavioral factors when conservative medical therapy is inadequate for symptom relief. The model represents a multidisciplinary approach to patient care which is described through case presentations. The results of research on the incidence of primary psychopathology in 53 children and 322 adults evaluated during a 3-year period for temporomandibular joint dysfunction and facial pain are also presented. It was found that children were more likely to be psychiatrically impaired (25%) than adults (7%). Children had a variety of psychiatric diagnoses including depression, conversion and adjustment disorders, overanxious behavior, and anorexia nervosa. The benefits of a multidisciplinary approach are discussed in terms of the efficacy of this coordinated treatment effort in ameliorating symptoms.

Biofeedback and relaxation-response training in the treatment of pediatric migraine.

To assess the efficacy of electromyographic biofeedback, relaxation-response training and pain behavior management as a treatment for pediatric migraine, we studied 18 children between the ages of eight and 12 years (mean = 10 X 1) in a prospective, randomized, controlled investigation. Six patients received all three treatment procedures, six received relaxation-response training and pain behavior management, and the remaining six constituted a waiting-list control group. All patients kept a record of their headaches for the 15-week study period and then for four weeks one year later. Following four weeks of baseline, the treatment groups completed nine one-hour treatment sessions in 11 weeks. Both treatment groups experienced a significant reduction in headache symptoms and were significantly improved compared to the waiting-list control group by the end of treatment. The treatment groups did not differ from each other in any of these comparisons. The reduction in headache symptoms in the treatment groups was maintained one year after treatment ended. These results suggest that relaxation-response training, with or without biofeedback training, combined with pain behavior management, is an effective alternative treatment for pediatric migraine.

Behavioral approaches to the management of chronic pain in children.

This article focuses on the basic premises underlying the behavioral assessment and treatment of chronic pain, particularly as they apply to the less distinct pain disorders of childhood, such as obscure headache and abdominal pain. Pain behavior management procedures, relaxation techniques, and biofeedback training are discussed in detail in reviewing recent research developments in this area.

Behavioral interventions and stress management training for hospitalized adolescents and young adults with cystic fibrosis.

Recent studies have demonstrated that the majority of cystic fibrosis (CF) patients are not at high risk for developing psychological problems. Clinical studies emphasizing the development of adaptive coping mechanisms in these patients have been suggested as a more appropriate line of research. The purpose of the present paper is to describe our experience in teaching various behavioral and stress management strategies to help CF patients. Behavioral counseling, relaxation training, and biofeedback have all been used with these patients to help them manage a number of problems more effectively. The predominant presenting problems have included elevated anxiety levels, sleeping difficulties, pain, and hyperventilation episodes. The typical treatment course with these patients is described and a case example is given to help elucidate the nature of behavioral interventions. Patient satisfaction ratings indicate that most patients view these techniques positively. Clinical observations suggest that the acquisition of behavioral coping skills may enhance the CF patient's perceived control of his/her situation, reduce the level of pain and anxiety, and enhance the quality of life.

Effects of naloxone on normals and chronically anxious patients.

A significant number of normal and chronically anxious subjects were able to discriminate subjectively between a session in which they received naloxone and one which they received a placebo. However, affective and physiological measures did not differ significantly between the drug and placebo sessions. The weakness of the response makes it unlikely that a naloxone-responsive endogenous opioid system is substantially involved in the regulation of anxiety. An unexpected finding from the placebo session data was that, despite increased forehead muscle tension and high self-ratings of distress, chronically anxious subjects showed consistently stable sympathetic activity under resting conditions and mild stress, indicating the existence of a subgroup of generalized anxiety patients with low autonomic reactivity.

Biofeedback treatment of fecal incontinence in patients with myelomeningocele.

Approximately six hours of biofeedback training was given to eight fecally incontinent children with myelomeningocele in order to establish bowel control. Their ages ranged from five to 15 years. The patients were shown a polygraph tracing of the external anal sphincter while they were being encouraged voluntarily to contract the sphincter when the rectum was distended with progressively larger volumes of air in a balloon. Seven of the eight patients showed normal sensation for rectal distension. Following this training period, five of the children had no incontinent periods, and two of these had discontinued enemas or suppositories. A sixth patient had an 80 per cent reduction in the frequency of incontinence. The remaining two did not benefit. At follow-up between 13 and 24 months later, four children were incontinent once a month or less often; two others were incontinent once per day, a considerable decrease from the pretraining period. The two children who failed to learn were still incontinent at follow-up. These results show that fecally incontinent patients with myelomeningocele can learn to evacuate normally or to reduce soiling after a relatively short period of biofeedback training.

Behavioral control of medicine compliance.

This study examined the effectiveness of several behavioral techniques on compliance of college students taking vitamin C on q.i.d. regimen. Compliance was assessed by a new technique using a variation of the urine tracer procedure designed specifically for this study. Subjects were provided vitamin C tablets, with three tablets per week containing phenazopyridine, a drug that produces a bright red-orange urine discoloration. Subjects were requested to indicate when urine discolorations occurred, and compliance was assessed by comparing the time of their report to the time predicted on the basis of the scheduled sequence of vitamin C and phenazopyridine tablets. Baseline compliance was assessed for 72 subjects over a three-week period, with the 40 most noncompliant subjects randomly assigned to four groups for Treatment I. The groups were: self-monitoring, taste, taste and self-monitoring, and a no-treatment control group. The self-monitoring procedure involved recording the time medicine was taken; the taste procedure involved providing the subjects with flavored tablets to increase the saliency of tablet taking; and the self-monitoring and taste procedure involved providing subjects with flavored tablets and asking them to record the flavor of each tablet they ingested. At the end of six weeks, half the subjects in each of these groups participated in response-cost procedures while the remaining subjects continued with their previous procedures. Response-cost procedures were implemented by returning a portion of the subjects' deposit only if a preset compliance criterion was met. Treatment II procedures were implemented for an additional three weeks. Results indicated the self-monitoring and taste plus self-monitoring procedures were superior during Treatment I. The implementation of response cost during Treatment II was associated with a marked improvement in compliance, independent of the history of noncompliance. The effects of the taste plus self-monitoring procedure were maintained during Treatment II and results obtained by this procedure were not significantly different from effects of response cost.