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Developing a Risk Prediction Model for Long-Term Physical and Psychological Functioning after Hematopoietic Cell Transplantation.

ABSTRACT: Hematopoietic cell transplantation (HCT) is associated with impaired physical and psychological functioning for some long-term survivors. A risk prediction model would help clinicians estimate their patients' physical and psychological functioning after HCT and determine when to refer to added supportive care when appropriate. The purpose of the present study was to develop risk prediction models for physical and psychological functioning in HCT survivors. This was a secondary analysis of data from a randomized controlled trial (NCT00799461) that included 3- to 10-year HCT survivors. Risk predictions for physical and psychological functioning were developed by using backward logistic regression. The models were internally validated using bootstrapping techniques. Regression coefficients were converted into easy-to-use risk scores. Finally, the sensitivity, specificity, and positive and negative predictive values of the total risk score were calculated. The analyses included 489 survivors, with a mean age of 45.6 (SD, 12.4) years; 47% were female and with a mean of 6.1 years (SD, 2.0) after transplantation. Younger age, higher body mass index (BMI), no or part-time work, more comorbid diseases, autologous transplantation, and chronic graft-versus-host disease predicted impaired physical functioning. Female gender, younger age, higher BMI, not living with a partner, autologous transplantation, and chronic graft-versus-host disease predicted impaired psychological functioning. Although both models had predictive value for long-term functioning, diagnostic accuracy was moderate. For the physical functioning receiver operating characteristic, area under the curve (AUC) after internal validation was .74 with sensitivity 51.9 and specificity 82.8 at the optimal cut-off. For psychological functioning, AUC after internal validation was .69 with sensitivity 83.3 and specificity 42.9 at the optimal cut-off. We conclude that it is possible to predict long-term physical and psychological functioning with readily accessible, mostly pretransplantation predictors. The accuracy of the risk prediction models can be improved for use in clinical practice, potentially by adding pretransplantation patient-reported functioning and comorbidities.

SUBMITTER: Braamse AM

PROVIDER: S-EPMC4753122 | biostudies-literature | 2016 Mar

REPOSITORIES: biostudies-literature

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Developing a Risk Prediction Model for Long-Term Physical and Psychological Functioning after Hematopoietic Cell Transplantation.

Braamse Annemarie M J AM Yi Jean C JC Visser Otto J OJ Heymans Martijn W MW van Meijel Berno B Dekker Joost J Syrjala Karen L KL

Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation 20151211 3

Hematopoietic cell transplantation (HCT) is associated with impaired physical and psychological functioning for some long-term survivors. A risk prediction model would help clinicians estimate their patients' physical and psychological functioning after HCT and determine when to refer to added supportive care when appropriate. The purpose of the present study was to develop risk prediction models for physical and psychological functioning in HCT survivors. This was a secondary analysis of data f ...[more]

PMID: 26685773

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Project description:Importance:Autologous hematopoietic stem cell transplantation (AHSCT) may be effective in aggressive forms of multiple sclerosis (MS) that fail to respond to standard therapies. Objective:To evaluate the long-term outcomes in patients who underwent AHSCT for the treatment of MS in a large multicenter cohort. Design, Setting, and Participants:Data were obtained in a multicenter, observational, retrospective cohort study. Eligibility criteria were receipt of AHSCT for the treatment of MS between January 1995 and December 2006 and the availability of a prespecified minimum data set comprising the disease subtype at baseline; the Expanded Disability Status Scale (EDSS) score at baseline; information on the administered conditioning regimen and graft manipulation; and at least 1 follow-up visit or report after transplant. The last patient visit was on July 1, 2012. To avoid bias, all eligible patients were included in the analysis regardless of their duration of follow-up. Data analysis was conducted from September 1, 2014 to April 27, 2015. Exposures:Demographic, disease-related, and treatment-related exposures were considered variables of interest, including age, disease subtype, baseline EDSS score, number of previous disease-modifying treatments, and intensity of the conditioning regimen. Main Outcomes and Measures:The primary outcomes were MS progression-free survival and overall survival. The probabilities of progression-free survival and overall survival were calculated using Kaplan-Meier survival curves and multivariable Cox proportional hazards regression analysis models. Results:Valid data were obtained from 25 centers in 13 countries for 281 evaluable patients, with median follow-up of 6.6 years (range, 0.2-16 years). Seventy-eight percent (218 of 281) of patients had progressive forms of MS. The median EDSS score before mobilization of peripheral blood stem cells was 6.5 (range, 1.5-9). Eight deaths (2.8%; 95% CI, 1.0%-4.9%) were reported within 100 days of transplant and were considered transplant-related mortality. The 5-year probability of progression-free survival as assessed by the EDSS score was 46% (95% CI, 42%-54%), and overall survival was 93% (95% CI, 89%-96%) at 5 years. Factors associated with neurological progression after transplant were older age (hazard ratio [HR], 1.03; 95% CI, 1.00-1.05), progressive vs relapsing form of MS (HR, 2.33; 95% CI, 1.27-4.28), and more than 2 previous disease-modifying therapies (HR, 1.65; 95% CI, 1.10-2.47). Higher baseline EDSS score was associated with worse overall survival (HR, 2.03; 95% CI, 1.40-2.95). Conclusions and Relevance:In this observational study of patients with MS treated with AHSCT, almost half of them remained free from neurological progression for 5 years after transplant. Younger age, relapsing form of MS, fewer prior immunotherapies, and lower baseline EDSS score were factors associated with better outcomes. The results support the rationale for further randomized clinical trials of AHSCT for the treatment of MS.

Dataset Information

Developing a Risk Prediction Model for Long-Term Physical and Psychological Functioning after Hematopoietic Cell Transplantation.

Publications

Developing a Risk Prediction Model for Long-Term Physical and Psychological Functioning after Hematopoietic Cell Transplantation.

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