Acute health-related quality of life in children undergoing stem cell transplant: I. Descriptive outcomes.

There has been little empirical documentation of the acute effects of bone marrow or stem cell transplant (BMT) on children. In the present study, the responses of 153 children undergoing BMT were assessed in a prospective, longitudinal design. Children were assessed at the time of admission for transplant, then underwent weekly assessments to week +6, followed by monthly assessment to month +6. Data were obtained both by parent report and patient report (for patients age 5 and up) using the BASES scales. The major findings are: (1) children undergoing BMT enter the hospital with an already heightened level of distress (defined by high levels of somatic symptoms and mood disturbance, and low levels of activity) that increases dramatically following conditioning, reaching a peak approximately 1 week following transplant; (2) this increased distress is transient, declining rapidly back to admission levels by week +4 to week +5, followed by a further decline to presumed basal levels by months 4-6; and (3) the trajectories of distress depicted by both parent and child report are remarkably similar, each providing confirmatory support for the validity of the findings. These findings confirm a number of widely held clinical impressions that had not previously been documented empirically, and point to the need for new interventions or more intensive approaches to supportive care aimed at reducing levels of distress during the acute phase of transplant.

Acute health-related quality of life in children undergoing stem cell transplant: II. Medical and demographic determinants.

Medical and demographic variables were examined as predictors of acute health-related quality of life (HRQL), specifically, somatic distress, mood disturbance and activity levels, during the period of bone marrow transplant (BMT) hospitalization, and the transition phase in the months following hospital discharge. The responses of 153 children undergoing BMT were assessed by both parent report and patient self-report in a prospective longitudinal design. Type of transplant, diagnosis, age, gender, and socio-economic status (SES) were examined as predictor variables of patient outcome. Type of transplant, patient age, and SES emerged as significant determinants of patient response. Children undergoing unrelated donor (MUD) transplants experiencing the highest levels of distress, followed by those undergoing matched-sibling BMT, while those undergoing autologous transplant experienced the lowest levels of distress. Younger patients experienced lower levels of distress and better HRQL than older children and adolescents. Although patients from different SES backgrounds appeared very similar at the time of hospital admission, those from lower SES backgrounds demonstrated greater distress and disturbance in HRQL subsequently, and throughout the first 6 months post BMT. These findings help to target specific subgroups of patients that may be in greater need of preventive interventions or more aggressive supportive care.

Cognitive and academic functioning in survivors of pediatric bone marrow transplantation.

PURPOSE: To evaluate cognitive and academic functioning in survivors of pediatric bone marrow transplants (BMTs) at 1 and 3 years after a BMT. PATIENTS AND METHODS: In a prospective, longitudinal design, patients underwent a comprehensive battery of neurocognitive measures before admission for transplantation and at 1, 3, and 5 years after a BMT. This article describes a cohort of 102 survivors with follow-up data available for 1 year after a BMT, including 54 survivors with follow-up available for 3 years. This represents the largest cohort of pediatric BMT survivors yet reported in a prospective study. RESULTS: In the cohort as a whole, there were no significant changes on global measures of intelligence (intelligence quotient [IQ]) and academic achievement at either 1 or 3 years after a BMT, despite adequate power to detect an IQ change of three points or greater. Likewise, performance on specific tests of neuropsychologic function remained stable. No significant differences were observed between patients whose conditioning regimen included total-body irradiation (TBI) and those whose did not. The primary predictor of neurocognitive outcome was patient age, with younger patients more likely to show declines over time. The subset of patients who were less than 3 years of age at the time of transplantation seemed to be particularly vulnerable to cognitive sequelae. CONCLUSION: The use of BMTs with or without TBI entails minimal risk of late neurocognitive sequelae in patients who are 6 years of age or older at the time of transplantation. However, patients who are less than 6 years of age at the time of transplantation, and particularly those less than 3 years of age, seem to be at some risk of cognitive declines.

Assessment of health-related quality of life in acute in-patient settings: use of the BASES instrument in children undergoing bone marrow transplantation.

The Behavioral, Affective and Somatic Experiences Scale (BASES) represents a set of tools for assessing aspects of health-related quality of life (HRQL) in patients undergoing active, intensive therapy. Separate versions have been developed for parent, nurse and patient reports. The scales were constructed to be sensitive to change and appropriate for repeated measures in longitudinal designs. We report preliminary results with these measures from a sample of 105 children undergoing bone marrow transplantation (BMT). Adequate reliability of the instruments is documented through measures of both internal consistency and cross-informant consistency. Several analyses provide evidence of the clinical validity of the measures. Repeated-measures ANOVAs indicated reliable patterns of change over time, with trajectories that conformed to a priori predictions. Discriminative validity was demonstrated through detection of significant differences in the predicted direction between patients undergoing allogeneic and autologous BMT. Additional evidence for validity comes from the very similar symptom trajectories in parent, nurse and patient reports. Differences between the BASES and other measures of HRQL are identified and alternative uses of the instruments are discussed.