HOME-BASED CONTINUOUS 24-HOUR DEFEROXAMINE VIA A PERIPHERALLY INSERTED CENTRAL CATHETER IN SEVERE AND REFRACTORY IRON OVERLOAD: A CONVINIENT AND EFFECTIVE APPROACH IN THALASSEMIA MAJOR PATIENTS
(Abstract release date: 05/19/16)
EHA Library. Dewedar H. 06/09/16; 133042; E1493
Dr. Hany Dewedar
Contributions
Contributions
Abstract
Abstract: E1493
Type: Eposter Presentation
Background
Severe iron overload leads to significant morbidity and mortality in β-thalassemia major (TM) patients. Intensive chelation is required in these patients to reduce iron to lower risk thresholds and avoid the development or worsening of end-organ dysfunction. Continuous intravenous deferoxamine (DFO) infusion in the inpatient setting remains the only management option in patients who fail to respond to high chelator doses, combination therapy, or those with symptomatic dysfunction requiring rescue therapy. The approach, however, may be associated with inconvenience and time lost in prolonged hospital admissions.
Aims
To evaluate the efficacy and safety of home-based continuous 24-hour DFO infusion via a peripherally inserted central catheter (PICC) in TM patients with severe and refractory iron overload.
Methods
This was a retrospective cohort study of TM patients attending our center between October 2013 and August 2015 who had received home-based continuous 24-hour DFO infusion via PICC. To receive such therapy, patients had to have no contraindication to PICC line insertion of DFO. They also had to have severe iron overload with evidence of no response to mono or combined therapy with subcutaneous DFO, defeiprone, or deferasirox. Severe iron overload was defined as a liver iron concentration (LIC) >15 mg/g, cardiac T2* <8 msec, or cardiac T2* 8-15 msec with any of the following: a left-ventricular ejection fraction (LVEF) <45% per echocardiogarphy, cardiac arrhythmia, compensated or decompensated clinical heart failure, or two or more endocrinopathies. The dose of DFO was 40 mg/kg titrated based on the index (<0.025) by Porter el al, administered with or without concomitant oral chelation.
Results
A total of 41 patients received home-based continuous 24-hour DFO infusion via PICC during the observation period (mean age 28.4 ± 5.6 years, 51% male). Among these, 9 (22.0%) discontinued therapy, 7 (17.1%) only had <6 months follow up, while 25 (61.0%) patients had >6 months follow up and were included in efficacy analysis. Seventeen (42%) received concomitant deferasirox and 21 (51%) received deferiprone. Reported adverse events in the 41 patients included: local reaction (n=17), renal tubular acidosis (n=6), arthritis (n=4), line thrombosis (n=4), line displacement (n=4), local infection (n=4), systemic infection (n=2), retinopathy (n-1). None of the patients required hospitalization, developed new endocrine complications or progressed to advanced cardiac disease and no treatment related mortality was observed.
In the efficacy cohort (n=25, mean age 28.2 ± 5.0 years, 36% male), patients had receive treatment for an average of 431 ± 137 days. There was a significant and remarkable decrease in mean serum ferritin level (10848 ± 5872 to 4095 ± 4116 ng/mL, p=0.0001) and LIC (37.5 ± 9.9 to 12.6 ± 11.0, p=0.001) and a significant increase in cardiac T2* (8.1 ± 3.5 msec to 12.4 ± 6.3 msec, p=0.002). Heart failure was reversed in 2 out of 3 patients and LVEF improved from a mean of 40% to 60%. Eight out of 9 insulin-dependent diabetic patients had insulin dose reduction (mean reduction 34.3 ± 0.2 Units), while one patient shifted to an oral hypoglycemic. All of the eight patients on oral hypoglycemic agents prior to treatment manged to stop it successfully.
Conclusion
Home-based continuous 24-hour DFO infusion via PICC is an effective treatment option in TM patients with severe iron overload that could not be controlled otherwise by other chelation regimens. The convenience of home-based therapy further promotes the role of this therapy in such high risk patients, although data from prospective clinical trials remain of merit.
Session topic: E-poster
Keyword(s): Iron chelation, Thalassemia, Treatment
Type: Eposter Presentation
Background
Severe iron overload leads to significant morbidity and mortality in β-thalassemia major (TM) patients. Intensive chelation is required in these patients to reduce iron to lower risk thresholds and avoid the development or worsening of end-organ dysfunction. Continuous intravenous deferoxamine (DFO) infusion in the inpatient setting remains the only management option in patients who fail to respond to high chelator doses, combination therapy, or those with symptomatic dysfunction requiring rescue therapy. The approach, however, may be associated with inconvenience and time lost in prolonged hospital admissions.
Aims
To evaluate the efficacy and safety of home-based continuous 24-hour DFO infusion via a peripherally inserted central catheter (PICC) in TM patients with severe and refractory iron overload.
Methods
This was a retrospective cohort study of TM patients attending our center between October 2013 and August 2015 who had received home-based continuous 24-hour DFO infusion via PICC. To receive such therapy, patients had to have no contraindication to PICC line insertion of DFO. They also had to have severe iron overload with evidence of no response to mono or combined therapy with subcutaneous DFO, defeiprone, or deferasirox. Severe iron overload was defined as a liver iron concentration (LIC) >15 mg/g, cardiac T2* <8 msec, or cardiac T2* 8-15 msec with any of the following: a left-ventricular ejection fraction (LVEF) <45% per echocardiogarphy, cardiac arrhythmia, compensated or decompensated clinical heart failure, or two or more endocrinopathies. The dose of DFO was 40 mg/kg titrated based on the index (<0.025) by Porter el al, administered with or without concomitant oral chelation.
Results
A total of 41 patients received home-based continuous 24-hour DFO infusion via PICC during the observation period (mean age 28.4 ± 5.6 years, 51% male). Among these, 9 (22.0%) discontinued therapy, 7 (17.1%) only had <6 months follow up, while 25 (61.0%) patients had >6 months follow up and were included in efficacy analysis. Seventeen (42%) received concomitant deferasirox and 21 (51%) received deferiprone. Reported adverse events in the 41 patients included: local reaction (n=17), renal tubular acidosis (n=6), arthritis (n=4), line thrombosis (n=4), line displacement (n=4), local infection (n=4), systemic infection (n=2), retinopathy (n-1). None of the patients required hospitalization, developed new endocrine complications or progressed to advanced cardiac disease and no treatment related mortality was observed.
In the efficacy cohort (n=25, mean age 28.2 ± 5.0 years, 36% male), patients had receive treatment for an average of 431 ± 137 days. There was a significant and remarkable decrease in mean serum ferritin level (10848 ± 5872 to 4095 ± 4116 ng/mL, p=0.0001) and LIC (37.5 ± 9.9 to 12.6 ± 11.0, p=0.001) and a significant increase in cardiac T2* (8.1 ± 3.5 msec to 12.4 ± 6.3 msec, p=0.002). Heart failure was reversed in 2 out of 3 patients and LVEF improved from a mean of 40% to 60%. Eight out of 9 insulin-dependent diabetic patients had insulin dose reduction (mean reduction 34.3 ± 0.2 Units), while one patient shifted to an oral hypoglycemic. All of the eight patients on oral hypoglycemic agents prior to treatment manged to stop it successfully.
Conclusion
Home-based continuous 24-hour DFO infusion via PICC is an effective treatment option in TM patients with severe iron overload that could not be controlled otherwise by other chelation regimens. The convenience of home-based therapy further promotes the role of this therapy in such high risk patients, although data from prospective clinical trials remain of merit.
Session topic: E-poster
Keyword(s): Iron chelation, Thalassemia, Treatment
Abstract: E1493
Type: Eposter Presentation
Background
Severe iron overload leads to significant morbidity and mortality in β-thalassemia major (TM) patients. Intensive chelation is required in these patients to reduce iron to lower risk thresholds and avoid the development or worsening of end-organ dysfunction. Continuous intravenous deferoxamine (DFO) infusion in the inpatient setting remains the only management option in patients who fail to respond to high chelator doses, combination therapy, or those with symptomatic dysfunction requiring rescue therapy. The approach, however, may be associated with inconvenience and time lost in prolonged hospital admissions.
Aims
To evaluate the efficacy and safety of home-based continuous 24-hour DFO infusion via a peripherally inserted central catheter (PICC) in TM patients with severe and refractory iron overload.
Methods
This was a retrospective cohort study of TM patients attending our center between October 2013 and August 2015 who had received home-based continuous 24-hour DFO infusion via PICC. To receive such therapy, patients had to have no contraindication to PICC line insertion of DFO. They also had to have severe iron overload with evidence of no response to mono or combined therapy with subcutaneous DFO, defeiprone, or deferasirox. Severe iron overload was defined as a liver iron concentration (LIC) >15 mg/g, cardiac T2* <8 msec, or cardiac T2* 8-15 msec with any of the following: a left-ventricular ejection fraction (LVEF) <45% per echocardiogarphy, cardiac arrhythmia, compensated or decompensated clinical heart failure, or two or more endocrinopathies. The dose of DFO was 40 mg/kg titrated based on the index (<0.025) by Porter el al, administered with or without concomitant oral chelation.
Results
A total of 41 patients received home-based continuous 24-hour DFO infusion via PICC during the observation period (mean age 28.4 ± 5.6 years, 51% male). Among these, 9 (22.0%) discontinued therapy, 7 (17.1%) only had <6 months follow up, while 25 (61.0%) patients had >6 months follow up and were included in efficacy analysis. Seventeen (42%) received concomitant deferasirox and 21 (51%) received deferiprone. Reported adverse events in the 41 patients included: local reaction (n=17), renal tubular acidosis (n=6), arthritis (n=4), line thrombosis (n=4), line displacement (n=4), local infection (n=4), systemic infection (n=2), retinopathy (n-1). None of the patients required hospitalization, developed new endocrine complications or progressed to advanced cardiac disease and no treatment related mortality was observed.
In the efficacy cohort (n=25, mean age 28.2 ± 5.0 years, 36% male), patients had receive treatment for an average of 431 ± 137 days. There was a significant and remarkable decrease in mean serum ferritin level (10848 ± 5872 to 4095 ± 4116 ng/mL, p=0.0001) and LIC (37.5 ± 9.9 to 12.6 ± 11.0, p=0.001) and a significant increase in cardiac T2* (8.1 ± 3.5 msec to 12.4 ± 6.3 msec, p=0.002). Heart failure was reversed in 2 out of 3 patients and LVEF improved from a mean of 40% to 60%. Eight out of 9 insulin-dependent diabetic patients had insulin dose reduction (mean reduction 34.3 ± 0.2 Units), while one patient shifted to an oral hypoglycemic. All of the eight patients on oral hypoglycemic agents prior to treatment manged to stop it successfully.
Conclusion
Home-based continuous 24-hour DFO infusion via PICC is an effective treatment option in TM patients with severe iron overload that could not be controlled otherwise by other chelation regimens. The convenience of home-based therapy further promotes the role of this therapy in such high risk patients, although data from prospective clinical trials remain of merit.
Session topic: E-poster
Keyword(s): Iron chelation, Thalassemia, Treatment
Type: Eposter Presentation
Background
Severe iron overload leads to significant morbidity and mortality in β-thalassemia major (TM) patients. Intensive chelation is required in these patients to reduce iron to lower risk thresholds and avoid the development or worsening of end-organ dysfunction. Continuous intravenous deferoxamine (DFO) infusion in the inpatient setting remains the only management option in patients who fail to respond to high chelator doses, combination therapy, or those with symptomatic dysfunction requiring rescue therapy. The approach, however, may be associated with inconvenience and time lost in prolonged hospital admissions.
Aims
To evaluate the efficacy and safety of home-based continuous 24-hour DFO infusion via a peripherally inserted central catheter (PICC) in TM patients with severe and refractory iron overload.
Methods
This was a retrospective cohort study of TM patients attending our center between October 2013 and August 2015 who had received home-based continuous 24-hour DFO infusion via PICC. To receive such therapy, patients had to have no contraindication to PICC line insertion of DFO. They also had to have severe iron overload with evidence of no response to mono or combined therapy with subcutaneous DFO, defeiprone, or deferasirox. Severe iron overload was defined as a liver iron concentration (LIC) >15 mg/g, cardiac T2* <8 msec, or cardiac T2* 8-15 msec with any of the following: a left-ventricular ejection fraction (LVEF) <45% per echocardiogarphy, cardiac arrhythmia, compensated or decompensated clinical heart failure, or two or more endocrinopathies. The dose of DFO was 40 mg/kg titrated based on the index (<0.025) by Porter el al, administered with or without concomitant oral chelation.
Results
A total of 41 patients received home-based continuous 24-hour DFO infusion via PICC during the observation period (mean age 28.4 ± 5.6 years, 51% male). Among these, 9 (22.0%) discontinued therapy, 7 (17.1%) only had <6 months follow up, while 25 (61.0%) patients had >6 months follow up and were included in efficacy analysis. Seventeen (42%) received concomitant deferasirox and 21 (51%) received deferiprone. Reported adverse events in the 41 patients included: local reaction (n=17), renal tubular acidosis (n=6), arthritis (n=4), line thrombosis (n=4), line displacement (n=4), local infection (n=4), systemic infection (n=2), retinopathy (n-1). None of the patients required hospitalization, developed new endocrine complications or progressed to advanced cardiac disease and no treatment related mortality was observed.
In the efficacy cohort (n=25, mean age 28.2 ± 5.0 years, 36% male), patients had receive treatment for an average of 431 ± 137 days. There was a significant and remarkable decrease in mean serum ferritin level (10848 ± 5872 to 4095 ± 4116 ng/mL, p=0.0001) and LIC (37.5 ± 9.9 to 12.6 ± 11.0, p=0.001) and a significant increase in cardiac T2* (8.1 ± 3.5 msec to 12.4 ± 6.3 msec, p=0.002). Heart failure was reversed in 2 out of 3 patients and LVEF improved from a mean of 40% to 60%. Eight out of 9 insulin-dependent diabetic patients had insulin dose reduction (mean reduction 34.3 ± 0.2 Units), while one patient shifted to an oral hypoglycemic. All of the eight patients on oral hypoglycemic agents prior to treatment manged to stop it successfully.
Conclusion
Home-based continuous 24-hour DFO infusion via PICC is an effective treatment option in TM patients with severe iron overload that could not be controlled otherwise by other chelation regimens. The convenience of home-based therapy further promotes the role of this therapy in such high risk patients, although data from prospective clinical trials remain of merit.
Session topic: E-poster
Keyword(s): Iron chelation, Thalassemia, Treatment
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