HCT-CI score may predict mortality for nonmalignant disease

 

SALT LAKE CITY – Scores of 3 or higher on the Hematopoietic Cell Transplantation Comorbidity Index (HCT-CI) are associated with an increased risk of posttransplant mortality in certain patients undergoing allogeneic HCT for nonmalignant disease, according to findings from a review of more than 4,000 patients.

The exception was in patients undergoing HCT for hemoglobinopathies, Larisa Broglie, MD, reported at the combined annual meetings of the Center for International Blood & Marrow Transplant Research and the American Society for Blood and Marrow Transplantation.

The findings of the study, which is the largest to date to validate the usefulness of the HCT-CI for risk assessment in HCT patients with nonmalignant disease, have important implications for patient counseling and decision making, said Dr. Broglie of the Medical College of Wisconsin, Milwaukee.

Of 4,083 children and adults who underwent a first allogeneic HCT for a nonmalignant disease between 2007 and 2014 and who had sufficient follow-up (median, 39 months), 61% had an HCT-CI score of 0, 20% had a score of 1-2, and 19% had a score of 3 or higher.

 

After adjustment for age, disease, donor, graft source, recipient cytomegalovirus status, and performance status, scores of 3 or greater were associated with an overall increased hazard ratio for poor survival (HRs of 1.33 for scores of 3-4 and 2.31 for scores of 5 or greater, vs. 1.0 and 1.127 for scores of 0 or 1-2, respectively), she said.

Patients with an HCT-CI score of 0 had estimated 2-year overall survival of 82.7%, compared with 80.3% for scores 1-2, 74.0% for scores 3-4, and 55.8% for scores of 5 or greater.

Patients included in this study were identified from the Center for International Blood & Marrow Transplant Research database. They ranged in age from under 1 year to 77 years but most were young; the median age was 9 years and 78% of patients were under age 20.

HCT was performed for acquired aplastic anemia in 33% of patients, immune deficiencies in 19%, hemoglobinopathies in 16%, bone marrow failure in 12%, histiocytic disorders in 11%, metabolic disease in 9%, or autoimmune disease in less than 1%, she said, noting that the most frequent comorbidities seen within the entire cohort were moderate pulmonary disease, hepatic disease, and infection requiring ongoing treatment.

The effect of HCT-CI score on survival was present regardless of conditioning intensity and graft-versus-host disease prophylaxis, but scores predicted mortality risk differently based on underlying disease, and different comorbidities predominated in each disease category, she said, explaining that this was apparent when patients were stratified by the seven represented nonmalignant disease categories to account for disease heterogeneity.

 

For example, HCT-CI score predicted mortality risk in patients with aplastic anemia (HRs of 1.00, 1.19, and 2.06 for scores of 0, 1-2, and 3 or greater, respectively), and in patients with immune deficiency (HRs of 1.00, 1.37, and 1.87 for scores of 0, 1-2, and 3 or greater, respectively), and the distribution of comorbidities in patients in these two disease categories was similar to that of the overall cohort.

However, HCT-CI score did not predict mortality risk in those undergoing HCT for hemoglobinopathies (HRs of 1.00, 0.46, and 0.59 for scores of 0, 1-2, and 3 or greater, respectively), Dr. Broglie said, noting that these patients had overall high survival rates regardless of HCT-CI scores, and they had comorbidities that differed from the overall cohort.

HCT is a curative treatment strategy for many patients with nonmalignant diseases but transplant-related mortality remains a concern, she said. While HCT-CI has been shown to be useful for discriminating the risks of nonrelapse and overall survival among patients with hematologic malignancies who undergo allogeneic HCT, its usefulness in patients undergoing HCT for nonmalignant diseases has been less clear.

The distinction is important, as patients with nonmalignant diseases have different pretransplant exposures and may have comorbidities specific to their underlying disease. Furthermore, transplantation approaches – including conditioning regimens and intensities – differ, she said.

 

“And the HCT-CI was developed to predict risk of nonrelapse mortality, but relapse in nonmalignant diseases can often be difficult to define,” she added.

The current findings demonstrate the value of the HCT-CI in nonmalignant diseases, and “offer the potential to intervene with transplantation prior to the onset of comorbidities, or with efforts to prevent comorbidities prior to transplantation,” she said, concluding that “future efforts could focus on further refining pretransplant risk assessment for patients with nonmalignant diseases, especially for patients with hemoglobinopathies and HCT-CI scores of less than 3.”

Dr. Broglie reported having no financial disclosures.

sworcester@frontlinemedcom.com

SOURCE: Broglie L et al. Abstract 16.

 

SALT LAKE CITY – Scores of 3 or higher on the Hematopoietic Cell Transplantation Comorbidity Index (HCT-CI) are associated with an increased risk of posttransplant mortality in certain patients undergoing allogeneic HCT for nonmalignant disease, according to findings from a review of more than 4,000 patients.

The exception was in patients undergoing HCT for hemoglobinopathies, Larisa Broglie, MD, reported at the combined annual meetings of the Center for International Blood & Marrow Transplant Research and the American Society for Blood and Marrow Transplantation.

The findings of the study, which is the largest to date to validate the usefulness of the HCT-CI for risk assessment in HCT patients with nonmalignant disease, have important implications for patient counseling and decision making, said Dr. Broglie of the Medical College of Wisconsin, Milwaukee.

Of 4,083 children and adults who underwent a first allogeneic HCT for a nonmalignant disease between 2007 and 2014 and who had sufficient follow-up (median, 39 months), 61% had an HCT-CI score of 0, 20% had a score of 1-2, and 19% had a score of 3 or higher.

 

After adjustment for age, disease, donor, graft source, recipient cytomegalovirus status, and performance status, scores of 3 or greater were associated with an overall increased hazard ratio for poor survival (HRs of 1.33 for scores of 3-4 and 2.31 for scores of 5 or greater, vs. 1.0 and 1.127 for scores of 0 or 1-2, respectively), she said.

Patients with an HCT-CI score of 0 had estimated 2-year overall survival of 82.7%, compared with 80.3% for scores 1-2, 74.0% for scores 3-4, and 55.8% for scores of 5 or greater.

Patients included in this study were identified from the Center for International Blood & Marrow Transplant Research database. They ranged in age from under 1 year to 77 years but most were young; the median age was 9 years and 78% of patients were under age 20.

HCT was performed for acquired aplastic anemia in 33% of patients, immune deficiencies in 19%, hemoglobinopathies in 16%, bone marrow failure in 12%, histiocytic disorders in 11%, metabolic disease in 9%, or autoimmune disease in less than 1%, she said, noting that the most frequent comorbidities seen within the entire cohort were moderate pulmonary disease, hepatic disease, and infection requiring ongoing treatment.

The effect of HCT-CI score on survival was present regardless of conditioning intensity and graft-versus-host disease prophylaxis, but scores predicted mortality risk differently based on underlying disease, and different comorbidities predominated in each disease category, she said, explaining that this was apparent when patients were stratified by the seven represented nonmalignant disease categories to account for disease heterogeneity.

 

For example, HCT-CI score predicted mortality risk in patients with aplastic anemia (HRs of 1.00, 1.19, and 2.06 for scores of 0, 1-2, and 3 or greater, respectively), and in patients with immune deficiency (HRs of 1.00, 1.37, and 1.87 for scores of 0, 1-2, and 3 or greater, respectively), and the distribution of comorbidities in patients in these two disease categories was similar to that of the overall cohort.

However, HCT-CI score did not predict mortality risk in those undergoing HCT for hemoglobinopathies (HRs of 1.00, 0.46, and 0.59 for scores of 0, 1-2, and 3 or greater, respectively), Dr. Broglie said, noting that these patients had overall high survival rates regardless of HCT-CI scores, and they had comorbidities that differed from the overall cohort.

HCT is a curative treatment strategy for many patients with nonmalignant diseases but transplant-related mortality remains a concern, she said. While HCT-CI has been shown to be useful for discriminating the risks of nonrelapse and overall survival among patients with hematologic malignancies who undergo allogeneic HCT, its usefulness in patients undergoing HCT for nonmalignant diseases has been less clear.

The distinction is important, as patients with nonmalignant diseases have different pretransplant exposures and may have comorbidities specific to their underlying disease. Furthermore, transplantation approaches – including conditioning regimens and intensities – differ, she said.

 

“And the HCT-CI was developed to predict risk of nonrelapse mortality, but relapse in nonmalignant diseases can often be difficult to define,” she added.

The current findings demonstrate the value of the HCT-CI in nonmalignant diseases, and “offer the potential to intervene with transplantation prior to the onset of comorbidities, or with efforts to prevent comorbidities prior to transplantation,” she said, concluding that “future efforts could focus on further refining pretransplant risk assessment for patients with nonmalignant diseases, especially for patients with hemoglobinopathies and HCT-CI scores of less than 3.”

Dr. Broglie reported having no financial disclosures.

sworcester@frontlinemedcom.com

SOURCE: Broglie L et al. Abstract 16.

 

SALT LAKE CITY – Scores of 3 or higher on the Hematopoietic Cell Transplantation Comorbidity Index (HCT-CI) are associated with an increased risk of posttransplant mortality in certain patients undergoing allogeneic HCT for nonmalignant disease, according to findings from a review of more than 4,000 patients.

The exception was in patients undergoing HCT for hemoglobinopathies, Larisa Broglie, MD, reported at the combined annual meetings of the Center for International Blood & Marrow Transplant Research and the American Society for Blood and Marrow Transplantation.

The findings of the study, which is the largest to date to validate the usefulness of the HCT-CI for risk assessment in HCT patients with nonmalignant disease, have important implications for patient counseling and decision making, said Dr. Broglie of the Medical College of Wisconsin, Milwaukee.

Of 4,083 children and adults who underwent a first allogeneic HCT for a nonmalignant disease between 2007 and 2014 and who had sufficient follow-up (median, 39 months), 61% had an HCT-CI score of 0, 20% had a score of 1-2, and 19% had a score of 3 or higher.

 

After adjustment for age, disease, donor, graft source, recipient cytomegalovirus status, and performance status, scores of 3 or greater were associated with an overall increased hazard ratio for poor survival (HRs of 1.33 for scores of 3-4 and 2.31 for scores of 5 or greater, vs. 1.0 and 1.127 for scores of 0 or 1-2, respectively), she said.

Patients with an HCT-CI score of 0 had estimated 2-year overall survival of 82.7%, compared with 80.3% for scores 1-2, 74.0% for scores 3-4, and 55.8% for scores of 5 or greater.

Patients included in this study were identified from the Center for International Blood & Marrow Transplant Research database. They ranged in age from under 1 year to 77 years but most were young; the median age was 9 years and 78% of patients were under age 20.

HCT was performed for acquired aplastic anemia in 33% of patients, immune deficiencies in 19%, hemoglobinopathies in 16%, bone marrow failure in 12%, histiocytic disorders in 11%, metabolic disease in 9%, or autoimmune disease in less than 1%, she said, noting that the most frequent comorbidities seen within the entire cohort were moderate pulmonary disease, hepatic disease, and infection requiring ongoing treatment.

The effect of HCT-CI score on survival was present regardless of conditioning intensity and graft-versus-host disease prophylaxis, but scores predicted mortality risk differently based on underlying disease, and different comorbidities predominated in each disease category, she said, explaining that this was apparent when patients were stratified by the seven represented nonmalignant disease categories to account for disease heterogeneity.

 

For example, HCT-CI score predicted mortality risk in patients with aplastic anemia (HRs of 1.00, 1.19, and 2.06 for scores of 0, 1-2, and 3 or greater, respectively), and in patients with immune deficiency (HRs of 1.00, 1.37, and 1.87 for scores of 0, 1-2, and 3 or greater, respectively), and the distribution of comorbidities in patients in these two disease categories was similar to that of the overall cohort.

However, HCT-CI score did not predict mortality risk in those undergoing HCT for hemoglobinopathies (HRs of 1.00, 0.46, and 0.59 for scores of 0, 1-2, and 3 or greater, respectively), Dr. Broglie said, noting that these patients had overall high survival rates regardless of HCT-CI scores, and they had comorbidities that differed from the overall cohort.

HCT is a curative treatment strategy for many patients with nonmalignant diseases but transplant-related mortality remains a concern, she said. While HCT-CI has been shown to be useful for discriminating the risks of nonrelapse and overall survival among patients with hematologic malignancies who undergo allogeneic HCT, its usefulness in patients undergoing HCT for nonmalignant diseases has been less clear.

The distinction is important, as patients with nonmalignant diseases have different pretransplant exposures and may have comorbidities specific to their underlying disease. Furthermore, transplantation approaches – including conditioning regimens and intensities – differ, she said.

 

“And the HCT-CI was developed to predict risk of nonrelapse mortality, but relapse in nonmalignant diseases can often be difficult to define,” she added.

The current findings demonstrate the value of the HCT-CI in nonmalignant diseases, and “offer the potential to intervene with transplantation prior to the onset of comorbidities, or with efforts to prevent comorbidities prior to transplantation,” she said, concluding that “future efforts could focus on further refining pretransplant risk assessment for patients with nonmalignant diseases, especially for patients with hemoglobinopathies and HCT-CI scores of less than 3.”

Dr. Broglie reported having no financial disclosures.

sworcester@frontlinemedcom.com

SOURCE: Broglie L et al. Abstract 16.