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Health-related quality of life (HRQL) in children with sickle cell disease and thalassemia following hematopoietic stem cell transplant (HSCT).

ABSTRACT: Little is known regarding the health-related quality of life (HRQL) trajectory of children with sickle cell disease or thalassemia ("hemoglobinopathies") following hematopoietic stem cell transplantation (HSCT).We serially evaluated the HRQL of 13 children with hemoglobinopathies who received HSCT during two prospective multi-center studies using the Child Health Ratings Inventories (CHRIs). The HRQL scores among children with hemoglobinopathies, as reported separately by the children and their parents were compared using repeated measures models to scores of a comparison group of children receiving HSCT for malignancies or severe aplastic anemia.The sample included 13 children with hemoglobinopathies (median age: 8 years, range 5-18) and 268 children in the comparison group (median age: 11 years, range 5-18). There were similar rates of early infection, chronic graft versus host disease, and all-cause mortality between the two groups. There was no significant difference in recovery to baseline scores for physical, emotional, and role functioning by 3 months for either group. Children with hemoglobinopathies had higher HRQL scores for physical (beta = 12, se = 5.5, P = 0.01) and baseline emotional functioning (beta = 11.6, se = 5.5, P = 0.03) than the comparison group. For all domains for both groups, parent reports demonstrated a nadir at 45 days with recovery to baseline by 3 months following transplant. Children's ratings were higher than those of their parents in both diagnostic groups.Children with hemoglobinopathies had higher physical and emotional functioning scores prior to HSCT and experienced a similar pattern of recovery to their baseline functioning by 3 months post-HSCT when compared to children receiving HSCT for acquired conditions.

SUBMITTER: Kelly MJ

PROVIDER: S-EPMC3319491 | biostudies-literature | 2012 Oct

REPOSITORIES: biostudies-literature

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Health-related quality of life (HRQL) in children with sickle cell disease and thalassemia following hematopoietic stem cell transplant (HSCT).

Kelly Michael J MJ Pennarola Brian W BW Rodday Angie Mae AM Parsons Susan K SK

Pediatric blood & cancer 20111219 4

<h4>Background</h4>Little is known regarding the health-related quality of life (HRQL) trajectory of children with sickle cell disease or thalassemia ("hemoglobinopathies") following hematopoietic stem cell transplantation (HSCT).<h4>Procedures</h4>We serially evaluated the HRQL of 13 children with hemoglobinopathies who received HSCT during two prospective multi-center studies using the Child Health Ratings Inventories (CHRIs). The HRQL scores among children with hemoglobinopathies, as reported ...[more]

PMID: 22183952

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Project description:PurposePediatric patients who undergo hematopoietic cell transplant (HCT) are at risk for neurocognitive impairments, which can impact quality of life. Given limited long-term studies, we aimed to characterize the late neurocognitive outcomes in a cohort of pediatric HCT survivors.MethodsEligible survivors (HCT at age < 21 year and ≥ 1 year post-HCT) completed a 60-question survey of neurocognitive function and quality of life, which included the Childhood Cancer Survivor Study Neurocognitive Questionnaire (CCSS-NCQ) and the Neuro-Quality of Life Cognitive Function Short Form (Neuro-QoL). Analyses of risk factors included univariate comparisons and multivariable logistic regression.ResultsParticipants (n = 199, 50.3% female, 53.3% acute leukemia, 87.9% allogeneic transplants) were surveyed at median age of 37.8 years (interquartile range [IQR] 28.5-48.8) at survey and median 27.6 years (IQR 17.0-34.0) from transplant. On the CCSS-NCQ, 18.9-32.5% of survivors reported impairments (Z score > 1.28) in task efficiency, memory, emotional regulation, or organization, compared with expected 10% in the general population (all p < 0.01). In contrast, survivors reported average Neuro-QoL (T score 49.6±0.7) compared with population normative value of 50 (p = 0.52). In multivariable regression, impaired Neuro-QoL (T score < 40) was independently associated with hearing issues (OR 4.97, 95% CI 1.96-12.6), history of stroke or seizure (OR 4.46, 95% CI 1.44-13.8), and sleep disturbances (OR 6.95, 95% CI 2.53-19.1).ConclusionsAlthough long-term survivors of pediatric HCT reported higher rates of impairment in specific neurocognitive domains, cognitive quality of life was perceived as similar to the general population. Subsets of survivors with certain co-morbidities had substantially worse neurocognitive outcomes.Implications for cancer survivorsWhile the long-term impact of pediatric HCT can include neurocognitive deficits, survivors report average cognitive quality of life.

Project description:AbstractPreliminary evidence from a series of 4 adults with sickle cell disease (SCD) suggests that hematopoietic stem cell transplant (HSCT) improves cerebral hemodynamics. HSCT largely normalizes cerebral hemodynamics in children with SCD. We tested the hypothesis in adults with SCD that cerebral blood flow (CBF), oxygen extraction fraction (OEF), and cerebral metabolic rate of oxygen (CMRO2) measured using magnetic resonance imaging, normalized to healthy values, comparing measurements from ∼1 month before to 12 to 24 months after HSCT (n = 11; age, 33.3 ± 8.9 years; 389 ± 150 days after HSCT) with age-, race- and sex-matched values from healthy adults without sickle trait (n = 28; age, 30.2 ± 5.6 years). Before transplant, 7 patients had neurological indications for transplant (eg, overt stroke) and 4 had nonneurological reasons for haploidentical bone marrow transplant (haplo-BMT). All received haplo-BMT from first-degree relatives (parent, sibling, or child donor) with reduced-intensity preparation and maintained engraftment. Before transplant, CBF was elevated (CBF, 69.11 ± 24.7 mL/100 g/min) compared with that of controls (P = .004). Mean CBF declined significantly after haplo-BMT (posttransplant CBF, 48.2 ± 13.9 mL/100 g/min; P = .003). OEF was not different from that of controls at baseline and did not change significantly after haplo-BMT (pretransplant, 43.1 ± 6.7%; posttransplant, 39.6 ± 7.0%; P = .34). After transplant, CBF and OEF were not significantly different from controls (CBF, 48.2 ± 13.4 mL/100 g/min; P = .78; and OEF, 39.6 ± 7.0%; P > .99). CMRO2 did not change significantly after haplo-BMT (pretransplant, 3.18 ± 0.87 mL O2/100 g/min; posttransplant, 2.95 ± 0.83; P = .56). Major complications of haplo-BMT included 1 infection-related death and 1 severe chronic graft-versus-host disease. Haplo-BMT in adults with SCD reduces CBF to that of control values and maintains OEF and CMRO2 on average at levels observed in healthy adult controls. The trial was registered at www.clinicaltrials.gov as #NCT01850108.

Dataset Information

Health-related quality of life (HRQL) in children with sickle cell disease and thalassemia following hematopoietic stem cell transplant (HSCT).

Publications

Health-related quality of life (HRQL) in children with sickle cell disease and thalassemia following hematopoietic stem cell transplant (HSCT).

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