scholarly journals Overall Survival in Acquired Pure Red Cell Aplasia in Adults Following Immunosuppressive Therapy: Preliminary Results from the Nationwide Cohort Study (PRCA2016)

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 2593-2593
Author(s):  
Naohito Fujishima ◽  
Makoto Hirokawa ◽  
Kenichi Sawada ◽  
Shinji Nakao ◽  
Yuji Yonemura ◽  
...  
Keyword(s):  
Cohort Study ◽  
Survival Time ◽  
Immunosuppressive Therapy ◽  
Research Funding ◽  
Chelation Therapy ◽  
Pharmaceutical Research ◽  
Iron Chelation ◽  
Iron Chelation Therapy ◽  
Significant Difference ◽  
Lgl Leukemia

Abstract Background. Idiopathic PRCA and secondary PRCA not responding to the treatment of the underlying diseases are generally treated by immunosuppressive therapy. Because of the rarity of this disease, there are few reports regarding the long-term outcome following immunosuppression. We previously conducted the nationwide study for chronic PRCA in Japan that had been diagnosed between 1990 and 2006 across 109 institutions, and collected the data on a total of 185 patients (PRCA2004/2006 study). This study revealed that poor response to induction therapy and relapse of anemia were associated with death. There was no significant difference in survival between idiopathic and secondary PRCA in the PRCA2004/2006 study cohort. Objective. In order to identify the adverse risk factors for survival in acquired PRCA following immunosuppression in a prospective way, we have conducted another nationwide cohort study for acquired PRCA in adults (PRCA2016 study). The primary endpoint is the overall survival (OS) and the secondary endpoints include efficacy of immunosuppression, causes of treatment failure and survival times with iron-chelation therapy. Methods. This is designed as a prospective longitudinal observational study. Between 2006 and 2014, 554 PRCA patients were registered in the hematological disorder registry managed by the Japanese Society of Hematology. We sent the first questionnaires to the physicians asking for collaboration with this study and the responses showed the potential size of cohort was 181 patients (Fig. 1). We then sent the second questionnaires to collect data regarding underlying disease, laboratory findings including CBC and leukocyte differentials, results of bone marrow examination, immunological and cytogenetics, efficacy of immunosuppression, iron-chelation therapy in refractory cases and outcome. The review boards of participating institutions and the ethical committee of the JSH approved this study and informed consent was obtained from the patients. Results. As of June 16, 2018, 103 patients were registered in this study. Fifty-two patients were classified as having idiopathic PRCA and 51 patients as having secondary PRCA, including 16 thymoma- and 5 large granular lymphocyte (LGL) leukemia-associated PRCA. Seventy-one patients were treated with immunosuppressive agents. The response rates of cyclosporine, predonisolone, cyclophosphamide, azathioprine and combination with more than one immunosuppressants were 84% (43/51), 89% (8/9), 100% (2/2), 0% (0/1), 60% (9/15), respectively (Fig. 2). The Kaplan-Meyer estimates of the survival revealed that the estimated median survival time in idiopathic PRCA was 6,430 days and that in secondary PRCA has not yet been reached (Fig. 3). Survival time was not significantly different between the two subtypes of PRCA. Twenty-two deaths were reported and the major causes of death were infection and heart failure. Discussion/Conclusion. The most common subtypes of chronic PRCA in Japan were idiopathic, thymoma-associated and LGL leukemia-associated PRCA and this result was consistent with our observation in the PRCA2004/2006 study. Cyclosporine and prednisolone were frequently chosen for the induction therapy resulting in favorable responses. Preferential use of these two agents might reflect the physician's concern about the toxicity of the cytotoxic drugs. The median survival time of idiopathic PRCA was quite similar to the results in the previous cohort study. No significant difference in survival between idiopathic and secondary PRCA has been confirmed by the present study. These two cohort studies clearly indicate that novel diagnostic and predictive biomarkers should be developed to segregate the good and poor responders to immunosuppressive therapy in PRCA. Disclosures Nakao: Novartis: Honoraria; Kyowa Hakko Kirin Co., Ltd.: Honoraria; Alexion Pharmaceuticals, Inc.: Consultancy, Honoraria. Yonemura:Alexion Pharma: Honoraria, Research Funding. Matsuda:GlaxoSmithKline K.K.: Honoraria; Novartis Pharma K. K.: Honoraria; Chugai Pharmaceutical Co, Ltd.: Honoraria; Kyowa Hakko Kirin Co, Ltd.: Honoraria; Sumitomo Dainippon Pharma Co., Ltd.: Honoraria; Nippon Shinyaku Co., Ltd.: Honoraria; Celgene Corporation: Honoraria; Alexion Pharmaceuticals, Inc.: Honoraria; Sanofi K.K.: Honoraria; Beckman Coulter K.K.: Honoraria. Kurokawa:Otsuka Pharmaceutical: Research Funding; Astellas Pharma: Research Funding; MSD: Honoraria, Research Funding; Pfizer: Research Funding; Eizai: Research Funding; Ono Pharmaceutical: Honoraria, Research Funding; Sumitomo Dainippon Pharma: Research Funding; Teijin Pharma: Research Funding; Nippon Sinyaku: Honoraria, Research Funding; Takeda Pharmaceutical: Research Funding; Chugai Pharmaceutical: Research Funding; Kyowa Hakko Kirin: Honoraria, Research Funding. Arai:Novartis: Research Funding. Mitani:Kyowa Hakko Kirin Co., Ltd.: Consultancy, Research Funding, Speakers Bureau; Bristol-Myesr Squibb: Research Funding, Speakers Bureau; Celgene: Speakers Bureau; Chugai: Research Funding; Astellas: Research Funding; Sumitomo Dainippon: Research Funding; Novartis: Research Funding; Toyama Chemical: Research Funding.

scholarly journals Red Blood Cell Transfusion Independence Following the Initiation of Iron Chelation Therapy in Myelodysplastic Syndrome.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 4844-4844
Author(s):  
Maha A Badawi ◽  
Linda M Vickars ◽  
Jocelyn M Chase ◽  
Heather A Leitch
Keyword(s):  
Platelet Count ◽  
Myelodysplastic Syndrome ◽  
Research Funding ◽  
Chelation Therapy ◽  
Ferritin Level ◽  
Iron Chelation ◽  
Iron Chelation Therapy ◽  
Transfusion Independence ◽  
Transfusion Requirements ◽  
Rbc Transfusion

Abstract Abstract 4844 Background Iron chelation therapy (ICT) is often used to treat iron overload (IOL) in patients (pts) requiring transfusion of red blood cells (RBC) for chronic anemia. In myelodysplastic syndrome (MDS), guidelines recommend consideration of ICT in pts with lower risk International Prognostic Scoring System (IPSS) and IOL as defined by a ferritin level >1000 ug/l; IOL related organ dysfunction; or receipt of ≥20 RBC units. During treatment of a pt with MDS and IOL with ICT, RBC transfusion requirement (TR) ceased. Here we report his course and review reported cases of RBC transfusion independence (TI) or decreased RBC TR in MDS pts receiving ICT. Methods The pt chart was reviewed and reported cases identified by PubMed search using the terms ‘MDS’ and ‘iron chelation’. The clinical characteristics and course of published cases were summarized. Case A 76 year (y) old man was referred in May 2004 for management of MDS diagnosed in 1997, when the white blood cell (WBC) count was 2.4 ×109/l; neutrophils, 0.7 ×109/l; hemoglobin (Hb), 133 g/l; platelets, 108 ×109/l. Bone marrow aspiration and biopsy showed refractory anemia (RA), karyotype analysis 46,X,-Y,+8, and the IPSS score was intermediate-1. The erythropoitin (epo) level was 148.3 mIU/ml and the stem cell assay showed no epo-independent colony growth. In 2004 the Hb dropped to 60 g/l prompting the initiation of RBC transfusion support. He required 3 RBC units every 4 weeks to maintain a Hb >90 g/l and complained of fatigue and functional limitation. Creatinine, bilirubin, TSH, reticulocyte count, B12 and folate levels were all normal. The ferritin level in 2004 was 1293 ug/l and 2197 ug/l in 2006. He declined ICT with deferoxamine (DFO) but in 2006 accepted deferasirox (DFX). He required several dose interruptions and adjustments for renal insufficiency; the current dose is 5mg/kg/d with a normal creatinine. Two months (mo) after starting ICT, the Hb increased spontaneously to 109 g/l and he has not required RBC transfusion since. The mean Hb since starting ICT was 122 g/l and the ferritin decreased to 1082 ug/l in 2009. The most recent neutrophil count was 3.5 ×109/l, platelets consistently clump and the MCV is unchanged at 120 fl. He reports excellent energy and an improved quality of life, and has remained clinically well and RBC transfusion independent to the present, 36 mo from the initiation of ICT. Literature review There are 18 published cases of MDS showing improvement in Hb with ICT; 9 became RBC transfusion independent. Characteristics of the 10 TI pts were: median age at MDS diagnosis 58 (range 18-74) y; male, n=5. MDS subtype: RA, n=5; RARS, n=2, RCMD, n=1; RAEB, n=2. IPSS (reported in 8): low, n=1; int-1, n=5; int-1 or 2, n=1; high, n=1. ICT was: DFO, n=7; DFX, n=3. Median time to RBC TI was 17.5 (1-24) mo and TI duration 13 (3-28) mo to date. Of pts who had decreased RBC transfusion requirements with ICT but did not achieve transfusion independence: median age (reported in 3) was 67 (45-78) y; gender (reported in 3) female, n=3; MDS subtype: RA, n=8; RAEB-t, n=1; IPSS: int-1, n=3; ICT: DFO, n=8; DFX, n=1. Median time to decreased TR was 14.4 (3-24) mo; median duration of decreased TR (reported in 3) 9 (6-32) mo; initial TR 50.9 (19.7-447) g Hb/mo; median decrease in TR 12.7 (0.1-88) g Hb/mo. In one report of 6 pts, 2 with pancytopenia showed improvement with ICT in WBC from 1.4 to 1.9 ×109/l (p<0.0001) and neutrophils from 0.51 to 0.94 ×109/l (p<0.001). The platelet count increased from 16.6 to 22.5 ×109/l (p<0.001) and 14.6 to 29.6 ×109/l (p<0.00001) within 3 mo and the MCV decreased significantly in 5 by a mean of 5.1 (2.1-11.7) fl, normalizing in 2. In a second report, neutrophils increased in 8 of 9 pts; in 4 the initial neutrophil count was <1 ×109/l, and platelet counts increased in 7 of 11 pts, in 4 the initial platelet count was <20 ×109/l. Conclusions In summary, our pt is the 19th patient with MDS reported to date in whom improved Hb followed the initiation of ICT; 9 had a decrease in RBC transfusion requirements, and RBC transfusion independence occurred in 10. The remarkable course of these pts adds to evidence that ICT may be of clinical benefit for selected patients with MDS and IOL. Although the improvement in WBC and platelet counts with ICT in some pts implies a suppressive effect of IOL on hematopoiesis that may be abrogated by ICT, the mechanism by which the effects of ICT on transfusion requirements occur, and the frequency with which they occur, remains an area for future investigation. Disclosures Off Label Use: This presentation discusses the use of iron chelation therapy deferoxamine and deferasirox in patients with myelodysplastic syndrome.. Vickars:Novartis Canada: Honoraria, Research Funding. Leitch:Novartis Canada: Honoraria, Research Funding, Speakers Bureau.

Estimating the Total Cost of Infused Iron Chelation Therapy.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 5576-5576 ◽  
Author(s):  
Marie-Pierre Desrosiers ◽  
Krista A. Payne ◽  
Jean-Francois Baladi
Keyword(s):  
Cohort Study ◽  
Resource Use ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Cost Data ◽  
Iron Chelation Therapy ◽  
Cell Disease ◽  
Total Cost ◽  
Unit Costs ◽  
The Cost

Abstract Background: Patients suffering from β-thalassemia or sickle cell disease require on-going blood transfusions. Chronic transfusion, however, results in iron overload, which if not removed by iron chelation therapy (ICT), causes organ damage. Deferoxamine (DFO) is currently the standard of care for ICT, but many patients do not adhere to therapy possibly because of the need for almost daily infusions lasting 8 to 10 hours each. Rationale: While the impact of current care on clinical and patient outcomes is generally understood, less is known about the total cost of DFO therapy. Objectives: To identify a complete set of cost items to inform the development of an ICT related Resource Use Questionnaire (RUQ) for administration in an international cohort study of the actual cost of ICT in practice; and to obtain a preliminary, literature-based estimate of total annual per patient costs of ICT. Methods: A search of the literature (EMB Reviews; Scirus and Ovid Medline (1996+); PubMed (1995+) was performed using the following key words: thalassemia, sickle cell disease, myelodysplastic syndrome, cost, iron chelation, Desferal, deferoxamine, resource use, reimbursement and compliance. Cost items were extracted from eligible studies to create an aggregated, composite set of ICT-related variables to which unit costs (2004/2005 USD) were applied. Results: Of 396 abstracts obtained, all but 96 were excluded because ICT cost data were lacking. Of those retained, only 4 studies (1 Israël;1 US;2 UK) reported ICT-related costs (1 lifetime;3 annual). Cost variables differed markedly among studies each focusing on some specific aspect. The application of unit costs to the composite list of ICT-related variables and associated resource use profiles reveal that total annual per patient ICT costs may be as high as $7,487 to $15,836 (£4,191 to £8,865) depending on age. The cost of DFO accounts for only 16%–31% of these estimated total costs, with the balance accounted for by other annual ancillary expenditures such as equipment and supplies, monitoring, and home health care services. Total costs could well be underestimated given that component lifetime costs such as DFO treatment complications, the clinical sequelae of poor adherence to DFO, and the indirect costs of lost productivity were not included. Cost estimates will be supplemented and validated at the time of abstract presentation by the resource use and unit cost data generated by the RUQ employed in the aforementioned international cohort study. Conclusions: Estimated total costs of ICT are substantial and well exceed the cost of DFO alone. A paucity of published data related to the total costs of ICT underscores the need for additional ICT cost data from actual practice to better understand the economic impact of novel ICT agents.

The Palatability and Tolerability of Deferasirox (Exjade®) Taken with Meals, Different Liquids, or Crushed and Added to Food

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 5155-5155
Author(s):  
Stuart L Goldberg ◽  
Patricia Giardina ◽  
Joan Parkhurst Cain ◽  
Deborah Chirnomas ◽  
Jason Esposito ◽  
...  
Keyword(s):  
Adverse Events ◽  
Serum Ferritin ◽  
Research Funding ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Thalassemia Major ◽  
Iron Chelation Therapy ◽  
Age Group ◽  
Baseline Serum ◽  
Off Label

Abstract Abstract 5155 Introduction: Deferasirox (Exjade®, Novartis Pharmaceuticals) is an oral iron chelator indicated for the treatment of transfusional iron overload. The recommended mode of administration is to be taken on an empty stomach in water, apple juice or orange juice ≥30 minutes before food. However, there have been post-marketing reports of discontinuation or reduced compliance of deferasirox secondary to palatability and gastrointestinal adverse events. Registration trials with deferasirox did not evaluate different food combinations in an attempt to maintain predictable plasma levels. Early single dose studies suggested that the bioavailability of deferasirox is increased when administered with or before meals, and is positively influenced by fat content, but is not affected by degree of dispersion nor type of liquid. Long-term pharmacokinetic and tolerability studies involving a food effect have not been conducted to date, and the ability of alternate methods of administration to improve patient compliance with iron chelation therapy is unknown. Method: This is an ongoing single-arm, open-label, multi-center study designed to evaluate the palatability, safety, tolerability and pharmacokinetics of deferasirox when administered with food, dispersed in any liquid of choice, or crushed and added to food. The patient population includes patients with transfusional hemosiderosis (minimum entry serum ferritin ≥500 μ g/L) aged >2 years with thalassemia major, sickle cell disease (SCD), low or intermediate (INT-1) risk MDS or other anemias, who are on, starting, or resuming treatment with deferasirox. The study began with a 1-month run-in phase with deferasirox dosed according to prescribing information, then a 3-month assessment phase where subjects could choose each week from 5 general administration options including with or without meals, in the morning or evening, crushed and added to a soft food, or mixed in a liquid of choice. Subject diaries are used to record the meal and method of administration at the end of each week. Palatability is assessed with a modified facial hedonic scale, with additional directed questions capturing gastrointestinal side effects. This is a data analysis of the run-in phase. Result: Target enrollment has been met with 65 patients. Baseline data on the first 58 subjects include 8 in the 2 to <10 years of age group (median 7.5 years; range 3–9); 42 in the 10 to <60 years of age group (median 18.5 years; range 10–48); and 8 in the ≥60 years of age group (median 74 years; range71-83). Underlying hematologic diagnoses included SCD (41%), thalassemia major (29%), MDS (12%) and other anemias (17%). Sixty-nine percent of subjects were receiving deferasirox prior to entering the study. The median baseline serum ferritin level was 2405 μ g/L (range 560–8660) and was distributed as shown in Table 1. The most frequent adverse events were diarrhea (19%) and nausea (9%) (Table 2), which were more common in MDS (P=0.23 and P<0.01, respectively). Conclusion: This ongoing trial (NCT00845871) is evaluating whether alternative modes of administration improve palatability and tolerability while maintaining safety. Preliminary data from the assessment phase (deferasirox taken with meals, different liquids, or crushed and added to food) will be presented at the meeting. Disclosures: Goldberg: Novartis Oncology: Consultancy, Honoraria, Research Funding, Speakers Bureau. Off Label Use: Exjade, iron chelation therapy, off-label method of administration. Giardina:Novartis: Research Funding. Parkhurst Cain:Novartis: Research Funding. Chirnomas:Novartis: Research Funding. Esposito:Novartis: Employment. Paley:Novartis: Employment. Vichinsky:Novartis: Consultancy, Research Funding, Speakers Bureau; Hemaquest: Consultancy, Membership on an entity's Board of Directors or advisory committees; Apotex: Consultancy, Research Funding.

Application of Self-Efficacy Theory in Adherence to Iron Chelation Therapy: A Single-Center Cross-Sectional Study

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 5284-5284
Author(s):  
Kevin H.M. Kuo ◽  
Richard Ward
Keyword(s):  
Family Support ◽  
Self Efficacy ◽  
Chelation Therapy ◽  
Care Center ◽  
Insurance Coverage ◽  
Iron Chelation ◽  
Comprehensive Care ◽  
Iron Chelation Therapy ◽  
Cross Sectional ◽  
Significant Difference

Abstract 5284 Introduction: Poor adherence to iron chelation therapy (ICT) in beta-Thalassemia Major (TM) is associated with increased risk of cardiac complications and endocrinopathies, and lower survival, with substantial cost to the patient and the health care system. Canada is unique in that several predictors of non-adherence (Financial barriers to medical care, cost of medication and inadequate follow-up) are minimized due to the presence of universal health care, governmental subsidies for medications for patients with chronic disease, and the availability of comprehensive care center for most of the thalassemia patients in the country. Also, the availability of Deferiprone (DFP) via compassionate release program since July 2004 provides an alternative to patients intolerant or having suboptimal response to Deferoxamine (DFO) or Deferasirox (DFX). We hypothesize that the absence of these barriers improve adherence in the Canadian thalassemic population. We also explored self-efficacy as a concept of adherence behavior in our patient population, defined as “individuals' personal beliefs regarding their capabilities to carry out a specific task to achieve a desired outcome” (Bandura, 1989). Methods: A cross-sectional survey was conducted in June and July 2011 at a regional comprehensive care center for transfusion-dependent thalassemia patients. We assessed the age, sex, education, employment status, insurance coverage, types and dosage of ICT, self-reported level of adherence, and side effects. We adapted the Medication Adherence Self-Efficacy Scale (MASES) to assess self-efficacy (Ogedegbe, 2003). Results: Survey return rate was 45% (46/103), with each type of ICT proportionally represented (P = 0.6401). Eight surveys were discarded due to incompletion and 38 were analyzed. Thirty-two patients were on single agent ICT (6 on DFO, 23 on DFX, 3 on DFP) and 6 patients were on combination treatment (1 on DFO+DFX; 3 on DFO+DFP; 2 on DFX+DFP). Median duration of iron chelation was more than 10 years. All patients had either government (n = 10) or workplace (n = 28) coverage. Twenty-three patients (61%) were self-described as completely adherent and 15 were not completely adherent. Mean level of adherence is 90% (SD 16%), similar to those reported in the literature (Trachtenberg et al., 2011), with no significant difference between the different types of ICT (P = 0.1085). Half of the non-adherent patients (8/15, 53%) miss 1 prescribed day of medication per week. There was no significant difference between adherent and non-adherent patients in age (P = 0.1484), sex (P = 0.3764), type of insurance coverage (P = 4752), family support (P = 0.7190), type of ICT (P = 0.0611), participation and satisfaction with the Exjade Patient Support Program (P = 1.000 and 0.3012 respectively), duration of chelation (P = 0.3951), rate of side effects (P = 0.4167), or feelings of depression (P = 0.4780). There was a trend towards differences in education level (P = 0.0565) and a higher proportion of professionals in the non-adherent group. The mean self-efficacy score of patients self-described as completely adherent was significantly higher than the non-completely adherent group (2.66 vs 1.93, P<0.0001). Discussion: In this self-reported survey of patients on ICT in a Canadian regional comprehensive care center, age, presence of family support, and feelings of depression were not found to be a significant predictor of poor adherence, unlike previous studies. This could be because previous studies only examined certain types of ICTs whereas the present study examined all forms of chelation. Small sample sizes of patients on DFO and DFP is the main limitation of the study. This is also the first known application of self-efficacy theory in explaining adherence to ICT. Further studies are required to examine the internal consistency and test-retest reliability of MASES in evaluating self-efficacy in adherence to ICT. Disclosures: Kuo: Novartis Canada: Research Funding. Off Label Use: Deferiprone is an unlicensed drug in Canada and USA. It is an oral iron chelator.

Final Results From the Multicenter Compact Study of Complications in Patients with Sickle Cell Disease and Utilization of Iron Chelation Therapy: A Retrospective Medical Records Review.

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 2106-2106
Author(s):  
Lanetta B Jordan ◽  
Patricia Adams-Graves ◽  
Julie Kanter-Washko ◽  
Patricia Ann Oneal ◽  
Francis Vekeman ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Resource Utilization ◽  
Medical Records ◽  
Research Funding ◽  
Index Date ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Complication Rates ◽  
Iron Chelation Therapy ◽  
Cell Disease

Abstract Abstract 2106 Introduction: Over the past few decades, lifespans of sickle cell disease (SCD) patients have increased; hence, they encounter multiple complications. Early detection, appropriate comprehensive care, and treatment may prevent or delay onset of complications. There is a gap in the literature describing the SCD complication rates, blood transfusion patterns, iron chelation therapy (ICT) use, and associated resource utilization in SCD patients ≥16 years old. This study contributes to addressing this gap. Method: Medical records of 254 SCD patients ≥ 16 were retrospectively reviewed between August 2011 and July 2012 at three US tertiary care centers (University of Tennessee: 117; Tulane University: 72; Howard University: 65). Data were collected from patient's first visit after age 16 (index date) until the earliest indication of death, loss to follow-up, or last patient record on file prior to the centers' IRB submission dates. Patients were classified into one of three cohorts based on cumulative units of blood transfused and history of ICT: <15 units of blood and no ICT (minimally transfused, Cohort 1 [C1]), ≥15 units of blood and no ICT (Cohort 2 [C2]), and ≥15 units of blood and receiving ICT (Cohort 3 [C3]). SCD complication rates were expressed as the number of SCD complications recorded from patient charts per patient per year (PPPY) and compared among cohorts using rate ratios (RRs). Results: Cohorts 1, 2, and 3 consisted of 69, 91, and 94 patients, respectively. Mean (range) age at index date was similar across cohorts (27 yrs [16–65]) and all patients were African American. Mean length of observation was shorter among patients in C1 (yrs, C1: 6.6; C2: 8.2; C3: 8.1). Post index date, patients in C1 received an average of 1 unit of blood PPPY (p<0.001 vs. C2 and C3), whereas patients in C2 and C3 received an average of 10 and 15 units PPPY (p=0.112), respectively. Among patients with serum ferritin (SF) assessment within 60 days before ICT (n=57), mean (median) SF level was 4,881 ng/mL (4,040). Across all three cohorts, the most common SCD complication was acute pain crisis (69.8%), followed by infection/sepsis (5.1%), leg ulcers (2.9%), and avascular necrosis (2.3%). The rate (95% CI) of any SCD complications was the highest in C2 at 3.02 PPPY (2.89–3.14), followed by 2.26 PPPY (2.16–2.37) in C3, and 1.66 PPPY (1.54–1.77) in C1 (Table 1). Among transfused patients (C2+C3), those receiving ICT were less likely to experience SCD complications than those who did not (RR [95% CI] C2 vs. C3: 1.33 [1.25–1.42]). Similar trends (RR [95% CI]) were observed in emergency room (ER) visits and hospitalizations associated with SCD complications (C2 vs. C3, ER: 1.94 [1.70–2.21]; hospitalizations: 1.61 [1.45–1.78]), but not in outpatient visits. Conclusion: Results from this study highlight the significant burden of complications and the associated healthcare resource utilization for SCD patients. The results suggest that among regularly transfused patients, those who received ICT were less likely to experience complications than those without ICT. However, transfusions are not necessary for all patients with SCD and patients with more complications may have started transfusion therapy earlier. Patients receiving ICT may also receive closer monitoring, which may help with early identification and intervention to delay or prevent the development of complications and improve outcomes. Disclosures: Jordan: Novartis Pharmaceuticals Corporation: Consultancy, Speakers Bureau. Oneal:Novartis Pharmaceuticals Corporation: Honoraria. Vekeman:Novartis Pharmaceuticals: Research Funding. Bieri:Novartis Pharmaceuticals Corporation: Research Funding. Sasane:Novartis Pharmaceuticals: Employment. Marcellari:Novartis Pharmaceuticals Corporation: Employment. Magestro:Novartis Pharmaceuticals: Employment. Gorn:Novartis Pharmaceuticals Corporation: Research Funding. Duh:Novartis Pharmaceuticals: Research Funding.

Deferasirox Is Associated with Reduced Mortality Risk in a Medicare Population with Myelodysplastic Syndromes

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 426-426 ◽  
Author(s):  
Amer M. Zeidan ◽  
Franklin Hendrick ◽  
Erika Friedmann ◽  
Steven D. Gore ◽  
Maria R. Baer ◽  
...  
Keyword(s):  
Renal Disease ◽  
Research Funding ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Time Varying ◽  
Iron Chelation Therapy ◽  
Transfusion Threshold ◽  
Endocrine Disease ◽  
Risk Of Death ◽  
Observation Period

Abstract Abstract 426 Background: While there is extensive evidence of the impact of iron chelation therapy (ICT) on clinical outcomes in thalassemia patients (pts), there is a paucity of data relevant to myelodysplastic syndromes (MDS). In this study, we examined the association between oral deferasirox (DFX) therapy and conditions potentially associated with iron overload [congestive heart failure (CHF) and endocrine disease (diabetes or thyroid)], drug side effects (renal disease), and overall mortality, among MDS pts in a U.S. Medicare cohort. Methods: MDS pts from 2005–2008 were identified using ICD-9 CM codes (1 inpatient or 2 outpatient) from the 100% Medicare claims database. Pts meeting a minimum transfusion threshold of 20 units packed RBC were considered “eligible” for ICT. The study cohort consisted of ICT-eligible pts enrolled in Medicare Part D, under which oral drug prescriptions are reimbursed. The observation period began the week (wk) after the RBC threshold was met and ended at incident diagnosis of a condition of interest, death, or end of study. Patients receiving parenteral ICT (deferoxamine) during the observation period were excluded. Patients with a history of the specific condition (CHF, endocrine disease, renal disease) were excluded from the outcome-specific sample, but were included in overall survival analysis. Onset of CHF or renal disease was based on the presence of 1 inpatient or 2 outpatient Medicare claims with ICD-9-CM diagnoses of each condition. Onset of diabetes or thyroid disease was measured based on new use of oral anti-diabetic or thyroid medications, respectively. Covariates in the outcome models included demographics (age, sex, race), ICD-9 coded MDS risk category at diagnosis, baseline indicators for co-morbid conditions, and time-varying exposure to RBC, erythropoiesis-stimulating agents (ESA), lenalidomide and hypomethylating agents (HMA). Marginal structural models (MSM) estimated the effects of cumulative DFX, controlling for anemia management and patient characteristics. The analysis adjusted for effects of time-varying health status on the probability of receiving DFX. Results: The cohort of 4,226 beneficiaries with MDS was 54% female, 90% white, with mean age of 77.7 years. MDS risk status was 16% lower/del5q, 5% higher, & 79% not otherwise specified. Common baseline comorbidities included CHF (46%), diabetes (18%), thyroid disease (21%), and renal disease (29%). Drug exposures prior to cohort entry included ESA (84%), HMA (19%) and lenalidomide (7%). Prior ICT was noted for 4.5% of patients. Patients were observed for a median of 35 weeks. Death occurred in 2496 (59%) of the cohort. DFX was used during the observation period by 544 (12%) patients, with mean duration of 29.2 weeks (median 20.5). Estimates from MSM indicate that each incremental week of DFX was associated with a decreased risk of death [hazard ratio (HR) 0.987; 95%CI 0.981–0.993]. The magnitude of risk reduction increased from HR 0.77 (95%CI 0.536–1.02) for patients receiving 14–26 weeks of DFX to HR 0.342 (95%CI 0.179–0.651) for patients with 53 or more weeks. Cumulative RBC units were associated with increased risk of death (HR 1.01, 95%CI 1.007–1.013 per unit), as were a baseline solid tumor diagnosis, renal and cardiac disease, and poor predicted performance status. After controlling for time-varying incidence of sepsis, bleeds, and cytopenias in MSM treatment propensity model, DFX use was not found to be associated with altered risk of CHF, endocrine or renal disease. Conclusions: This is the first large population-based Medicare study evaluating the association between DFX use in older MDS patients and sequelae of iron overload and mortality. The decrease in risk of death was proportional to the duration of therapy. Application of MSM addresses the role of observed time-varying confounders between treatment and outcomes, while permitting us to assess the duration-outcome relationships. The MSM analysis cannot address potential confounding by unobserved factors that may influence physician selection of patients for ICT. Nonetheless, this retrospective analysis controls for bias associated with observed patient health status, and lends significant support to a positive association of oral iron chelation therapy with decreased mortality in MDS patients with a minimum transfusion threshold, and ongoing transfusion needs. Disclosures: Gore: Celgene Corporation: Consultancy, Research Funding. Baer:Novartis, Inc.: Research Funding; Celgene, Inc.: Research Funding. Sasane:Novartis Pharmaceuticals: Employment. Paley:Novartis: Employment. Davidoff:GlaskoSmithKline: Research Funding; National Institutes of Health: Research Funding; Celgene: Research Funding; Novartis: Research Funding.

High Resource Utilizers From The Multicenter Compact Study Of Complications In Patients With Sickle Cell Disease and Utilization Of Iron Chelation Therapy

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 982-982
Author(s):  
Lanetta B Jordan ◽  
Patricia Adams-Graves ◽  
Julie Kanter-Washko ◽  
Patricia A Oneal ◽  
Medha Sasane ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Research Funding ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Iron Chelation Therapy ◽  
Cell Disease ◽  
High Utilizers ◽  
Ed Visits ◽  
High Utilizer ◽  
High Utilization

Abstract Introduction While treating patients (pts) with sickle cell disease (SCD) can be costly, costs are not evenly distributed across pts; rather, a minority of pts accounts for a majority of costs. Identifying those pts who consume a disproportionately large share of healthcare resources can assist payers and providers in directing appropriate and targeted interventions to deliver better pt care with lower costs. The objective of this study was to understand characteristics of pts who have increased utilization of inpatient (IP) and emergency department (ED) resources in a population of SCD pts ≥16 years old. Method Medical records of 254 SCD pts ≥16 years old were retrospectively reviewed between 8/2011 and 7/2012 at three US tertiary care centers. The high utilization threshold was derived from the literature and defined as pts with ≥ 5 days of IP+ED care (assuming 1 day/ED visit) for SCD-related complications per year (high utilizer group). Pts were also classified into cohorts based on cumulative blood transfusion units and use iron chelation therapy (ICT): <15 units, no ICT (Cohort 1 [C1]), ≥15 units, no ICT (Cohort 2 [C2]), and ≥15 units, with ICT (Cohort 3 [C3]). SCD complication rates were expressed as the number of SCD complications per pt per year (PPPY); rate ratios (RRs) were used for cohort comparisons. A logistic regression was used to identify risk factors associated with high utilization of IP+ED care. Results Of the 254 pts (C1: 69, C2: 91, C3: 94), 30% (n =76) were classified as high utilizers (C1: 14 [18.4%], C2: 37 [48.7%], C3: 25 [32.9%]). Patients in the high utilizer group were younger (median [range] (21 years old [16-65], vs. 23 years old [16-59]) and had shorter follow-up (4.2 years [0.6-23.9], vs. 5.4 years [0.5-33.3]) compared to the rest of the sample. Those in the high utilizer group accounted for 68% of all SCD-related complications and over 88% of all IP+ED days for treatment of these complications. Similar to the rest of the sample, pain (81%) and infection (7%) were the two key complications seen in this high utilizer group. The rate of IP +ED days was significantly higher among the high utilizer group with 16.63 [16.28-16.99] IP+ED days PPPY compared to 0.89 [0.84-0.94] PPPY for other pts. Similarly, the high utilizer group had 4.58 [95% CI: 4.39-4.76] IP+ED visits PPPY, compared to 0.34 [0.31-0.37] visits PPPY for other pts (Table). Among regularly transfused pts (C2+C3) in the high utilizer group, those who received ICT had lower rates of IP+ED visits (C2 vs. C3 rate ratio [RR] [95% CI]: 1.31[1.20-1.44]), IP+ED days (C2 vs. C3 RR: 1.30 [1.24-1.36]), and readmission to IP+ED settings within 30 days (1.70 [1.49-1.93]) compared with those who did not (Table). History of infections (odds ratio: 7.45, p<0.0001) was associated with an increased risk of high utilization of IP+ED care. Conclusion Results from this study show that a relatively small fraction of SCD pts account for the majority of IP+ED visits. Moreover, among regularly transfused pts identified as high utilizers, those who received ICT had lower rates of IP+ED utilization than those who did not. Pts receiving ICT may also receive closer monitoring, which may help with early identification and intervention to delay or prevent the development of complications and improve outcomes. Closer management of pts with SCD, especially those at risk of becoming high utilizers, is critical to lowering IP+ED utilization and reducing the overall costs of care. Disclosures: Jordan: Novartis Pharmaceuticals Corporation: Consultancy. Adams-Graves:Analysis Group, Inc.: Research Funding. Kanter-Washko:Analysis Group, Inc.: Research Funding. Oneal:Novartis Pharmaceuticals Corporation: Honoraria; Analysis Group, Inc.: Research Funding. Sasane:Novartis Pharmaceuticals: Employment. Vekeman:Novartis Pharmaceuticals: Research Funding. Bieri:Novartis Pharmaceuticals Corporation: Research Funding. Marcellari:Novartis Pharmaceuticals Corporation: Employment. Magestro:Novartis Pharmaceuticals: Employment. Adams:Novartis Pharmaceuticals Corporation: Research Funding. Duh:Novartis Pharmaceuticals: Research Funding.

Clinical Parameters in 391 Iron-Overloaded Patients with Lower-Risk MDS Enrolled in a Prospective, Non-Interventional Multicenter Registry.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 4834-4834
Author(s):  
Guillermo Garcia-Manero ◽  
Billie J. Marek ◽  
Roger M. Lyons ◽  
Noelia Martinez-Lopez ◽  
Carole Paley ◽  
...  
Keyword(s):  
Iron Overload ◽  
Board Of Directors ◽  
Serum Ferritin ◽  
Lower Risk ◽  
Research Funding ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Iron Chelation Therapy ◽  
Advisory Committees ◽  
The Impact

Abstract Abstract 4834 Introduction Despite recent improvements in therapies for patients with myelodysplastic syndromes (MDS), 60–80% will require continuing packed red cell blood (pRBC) transfusions for prolonged periods. Complications resulting from the iron burden may, therefore, become clinically significant for many patients during the course of their disease. Patients with lower-risk MDS have a greater chance of developing the long-term toxicity of iron overload because of their prolonged survival, and are more likely to benefit from effective iron chelation therapy. This report describes data from a registry designed to study the impact of iron overload and iron chelation therapy on organ function and survival in patients with lower-risk MDS. Methods This is an ongoing, prospective, non-interventional, multicenter 5-year registry in 107 US centers, enrolling 600 patients (aged ≥18 years) with lower-risk MDS (by WHO, FAB and/or IPSS criteria) and transfusional iron overload (defined as serum ferritin ≥1000 μg/L and/or having received ≤20 cumulative pRBC units and/or an ongoing transfusion requirement ≥6 units every 12 weeks). Follow-up will be performed at least every 6 months for a maximum of 60 months or until death. Recommended assessments include serum ferritin, creatinine, calculated creatinine clearance, echocardiograms, and endocrine and hematological status. Results As of May 31 2009, 391 patients have enrolled in the registry. Demographic data are available from 389 patients. Median age: 74.4 years (range 21–99); male: 218, female: 171; ethnicity: 331 Caucasian (85%), 25 African-American (6%), 24 Hispanic (6%), five Asian (1%), two Native American (0.5%), and two other (0.5%). The median time since diagnosis (n=385) was <3 years in 217 patients (56%); ≥3–<5 years in 72 (19%); ≥5–<7 years in 48 (12%); and ≥7 years in 48 (12%). The MDS classification of the patients by WHO, FAB and IPSS, as well as patients' serum ferritin and transfusion burden, are summarized in the table. The most frequent concomitant conditions classified by organ (n=384 patients) were: 205 (53%) patients with vascular, 160 (42%) endocrine, and 171 (45%) cardiac dysfunction. At registry entry, 249 patients were receiving erythropoietin; 61 granulocyte colony stimulating factor; seven hydroxyurea; 25 thalidomide (Thalomid); 147 5-azacytidine (Vidaza); 95 lenalidomide (Revlimid) and 90 decitabine (Dacogen). 137 of 391 (35%) patients were on iron chelation therapy at study entry: 34 (9%) received deferoxamine for mean and median treatment durations of 803 and 383 (range 1–4386) days, respectively, while 117 (30%) received deferasirox for mean and median durations of 488 and 396 (9–1269) days, respectively. Calculated creatinine clearance was normal (>80 mL/min) in 37 (9%) patients; mildly abnormal (51–80 mL/min) in 30 (8%); and moderately abnormal (30–50 mL/min) in nine (2%) patients. Conclusions These baseline data indicate the demographic distribution as well as the co-morbidities associated with lower-risk MDS patients. In spite of recent guidelines, fewer than 50% of iron-overloaded patients are receiving any iron chelation treatment, despite the presence of cardiac, vascular and endocrine concomitant conditions in 40-54% of patients. Recent retrospective data highlights the impact of chelation on mortality in lower-risk MDS patients. This ongoing registry will prospectively assess the impact of iron chelation on survival and organ function in iron-overloaded patients with lower-risk MDS. Disclosures Lyons: Novartis: Research Funding; GlaxoSmithKline: Consultancy, Research Funding; Johnson & Johnson: Consultancy, Honoraria, Research Funding; Celgene: Consultancy, Research Funding; Amgen: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Genzyme: Research Funding. Martinez-Lopez:Novartis Pharmaceuticals: Employment. Paley:Novartis Pharmaceuticals: Employment, Equity Ownership. Greenberg:Amgen: Consultancy, Research Funding; Celgene: Consultancy, Membership on an entity's Board of Directors or advisory committees; Novartis: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding.

Improved Patient-Reported Outcomes with a Film-Coated Versus Dispersible Tablet Formulation of Deferasirox: Results from the Randomized, Phase II E.C.L.I.P.S.E. Study

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 850-850 ◽  
Author(s):  
Ali T. Taher ◽  
Raffaella Origa ◽  
Silverio Perrotta ◽  
Alexandra Kouraklis ◽  
Giovan Battista Ruffo ◽  
...  
Keyword(s):  
Iron Overload ◽  
Research Funding ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Iron Chelation Therapy ◽  
Fasting State ◽  
Once Daily ◽  
End Of Treatment ◽  
Gi Symptoms ◽  
Dispersible Tablet

Abstract Background : Patients (pts) with hematological disorders such as transfusion-dependent thalassemia (TDT) and myelodysplastic syndromes (MDS) require long-term supportive therapy with iron chelation therapy (ICT) to remove excess iron and prevent organ failure. Adherence to ICT can affect survival, so a well-tolerated and effective chelation regimen is required. The once-daily dispersible tablet (DT) formulation of the iron chelator deferasirox (DFX) has been available since 2005, offering an alternative to parenteral deferoxamine, yet barriers remain to pt adherence including the need to take the drug in a fasting state, palatability and GI tolerability. A DFX film-coated tablet (FCT) was developed that can be taken orally, once-daily with or after a light meal, and is potentially less burdensome to pts than the DT formulation. Here we report pt-reported outcomes, during the 24-week (wk) study. Methods :ICT-naïve or pre-treatedpts aged ≥10 yrs, requiring ICT at DFX DT ≥30 mg/kg/day (TDT) or ≥20 mg/kg/day (MDS), with serum ferritin (SF) >1000 ng/mL, were enrolled. ICT-naïve pts received either DFX DT 20 mg/kg/day or DFX FCT 14 mg/kg/day. ICT pre-treated pts received a DT or FCT dose equivalent to their pre-washout dose. Dose was adjusted based on SF and investigator's judgment after wk 4 for ICT-naïve pts and after 3 months for ICT pre-treated pts (±5-10 mg/kg/day, maximum 40 mg/kg/day [DT]; ±3.5-7 mg/kg/day, maximum 28 mg/kg/day [FCT]); dose adjustments for safety reasons permitted at any time. The modified satisfaction with iron chelation therapy (modified SICT; 5-point response scales to assess adherence (six questions), satisfaction/preference (two questions), and concern (three questions) domain scores) and palatability (taste, aftertaste, ability to consume medicine, perception of medicine) questionnaires were completed at wks 2, 3, 13, and end of treatment. The GI symptom diary consisted of five items (pain in your belly, nausea, vomiting, constipation, diarrhea) rated on an 11-point scale and was completed daily. Results: In total, 173 pts were enrolled; 87 were treated with FCT and 86 were treated with DT. Completion rates for questionnaires (~80% at the start reducing to ~70% by wk 24) and GI daily diary (~70% at the start reducing to ~35% by wk 24) were similar for each formulation. Throughout the 24-wk study period, FCT pts consistently reported greater adherence (attributable to: easier to remember to take medication, thinking less about stopping medication, instructions from doctor followed more closely, medication easier to take, less bothered by time taken to prepare medication and waiting time before eating), greater satisfaction/preference (in general and with administration of medicine), and fewer concerns (attributable to: less worry about not swallowing enough medication, fewer limitations in daily activities, less concern about side effects), than DT pts (Figure 1). Pts assessed which medication for iron overload they would like best, choosing between: FCT, DT, powder to sprinkle on food, and 'I don't know'. At end of treatment, 53/60 (88.3%) evaluable pts receiving FCT indicated that they preferred the FCT and 41/63 (65.1%) evaluable pts receiving DT indicated they would prefer FCT. FCT pts reported higher satisfaction on palatability scores than DT pts (Figure 1), reporting no taste or aftertaste and that they are able to swallow the full amount of medicine with the right amount of liquid. The difference in score between DT and FCT was >1 point (minimal important difference) for all domains and palatability at most visits, indicating a clinically meaningful difference between formulations. Overall GI summary scores were low for both formulations indicating pts experience very little trouble/concern associated with GI symptoms; DT pts reported more trouble/concern than those receiving FCT (Figure 2). Conclusions: These results show a clear preference in favor of DFX FCT in all domains for the modified SICT. Pts were satisfied with their medicine during the study period and more pts were satisfied with DFX FCT compared with DT at all visits. DFX FCT offers pts an improved formulation that does not require administration in a fasting state, has better palatability, and very little concern associated with GI tolerability. Enhanced pt satisfaction with the new DFX FCT formulation may improve adherence, thereby, reducing iron overload-related complications. Disclosures Taher: Novartis: Honoraria, Research Funding; Celgene: Research Funding. Origa:Novartis: Honoraria; Apopharma: Honoraria. Kouraklis:Novartis: Honoraria; Amgen: Honoraria; Janssen: Honoraria; Gilead: Consultancy; Roche: Consultancy; Celegene: Consultancy. Kattamis:Novartis: Honoraria, Research Funding; ApoPharma: Honoraria. Cortoos:Novartis: Employment. Huang:Novartis: Employment. Weill:Novartis: Employment. Herranz:Novartis: Employment. Porter:Bluebird Bio: Consultancy; Agios Pharmaceuticals: Consultancy, Honoraria; Celegene: Consultancy; Novartis: Consultancy, Honoraria, Research Funding.

scholarly journals Adherence to Iron Chelation Therapy and Associated Healthcare Resource Utilization and Costs in Medicaid Patients with Sickle Cell Disease

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 2177-2177
Author(s):  
Francis Vekeman ◽  
Medha Sasane ◽  
Wendy Y Cheng ◽  
Agnihotram V Ramanakumar ◽  
Jonathan Fortier ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Emergency Room ◽  
Resource Utilization ◽  
Sickle Cell ◽  
Research Funding ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Cost Of Care ◽  
Iron Chelation Therapy ◽  
Cell Disease

Abstract Introduction: While adherence to iron chelation therapy (ICT) is critical for successful iron overload (IO) treatment among patients with sickle cell disease (SCD), published data indicate it is often suboptimal. Deferoxamine (DFO) and Deferasirox (DFX) are ICTs indicated for the treatment of chronic IO in patients with SCD. Lack of patient adherence may impact patient outcomes and increase cost of care. This study evaluated the economic burden of ICT non-adherence in patients with SCD from the state Medicaid program’s perspective. Methods: Patients with SCD were identified from Florida, Iowa, Kansas, Mississippi, Missouri, and New Jersey (1997-2013) state Medicaid programs. Patients were required to have ≥1 ICD-9 diagnosis code for SCD (282.6), ≥1 prescription for DFO or DFX, and ≥6 months of continuous enrollment prior to the 1st DFO/DFX prescription (index date), which was defined as the baseline period. Adherence was estimated using the medication possession ratio (MPR), defined as the sum of the days of medication supply divided by the number of days between 1st and last prescription fill plus the days of supply of the last fill; a threshold of ≥0.80 was used to define optimal adherence. All-cause and SCD-specific resource utilization per-patient-per-month (PPPM) was assessed using cumulative rates, accounting for all visits observed, and compared between adherent and non-adherent patients using cumulative rate ratios (CRR). All-cause and SCD-specific healthcare costs were computed using mean cost PPPM. Regression models adjusting for baseline characteristics were used to assess resource utilization and cost differences between adherent and non-adherent patients. Results: A total of 846 eligible patients with SCD were included with 77 in DFO-only, 686 in DFX-only), and 83 in DFO/DFX switch cohort. Mean (SD) MPR was 0.68 (0.27) for DFO-only patients and 0.75 (0.26) for DFX-only patients (p<0.05). Among all users of ICT, 409 (48.3%) were considered adherent. Adherent patients were slightly younger (19 vs. 21 years, p=0.003) than non-adherent patients. Rates of transfusions were comparable between the two groups (mean [SD] transfusions PPPM, adherent: 0.41 [0.47]; non-adherent: 0.40 [0.54], p=0.456) at baseline. The adjusted rate of all-cause IP visits PPPM was lower in adherent versus non-adherent patients (CRR=0.87 [95% CI: 0.83, 0.91]; p<0.001). The adjusted rates of all-cause outpatient (OP) visits (1.10 [1.08, 1.13], p<0.001) and ER visits (1.06 [1.01, 1.10], p=0.010) PPPM of adherent patients were higher in adherent patients than those in non-adherent patients. A similar trend was observed in SCD-specific resource utilization except for rates of ER visits, which were similar between cohorts. From cost perspective, total all-cause and SCD-specific costs were lower in adherent versus non-adherent patients primarily due to lower IP costs (Table 1). SCD-specific ER and OP costs were similar in both cohorts. All-cause pharmacy costs were higher in adherent versus non-adherent patients. Conclusion: Published studies have reported low adherence to ICT, and a similar trend was found in this study. Adherent patients were observed to have less frequent hospitalizations and lower overall and SCD-specific IP costs compared to non-adherent patients. It should be noted that the rate of OP visits was higher in the adherent patients compared to non-adherent patients suggesting that adherent patients may be more closely monitored potentially resulting in better overall patient management and fewer hospitalizations. Additional analyses are needed to explore differences between adherent and non-adherent patients. Table 1 Costs PPPM Adherent patients (N=409) [A] Non-adherent patients (N=437) [B] Adjusted cost difference[A] – [B] P -value All-cause, mean [SD] $4,766 [$4,388] $5,304 [$4,725] -$724 0.072 Inpatient $1,911 [$3,647] $2,996 [$4,439] -$947 0.016 Emergency room $27 [$87] $40 [$88] -$203 0.104 Outpatient $580 [$697] $485 [$617] $49 0.500 Pharmacy $2,248 [$1,949] $1,783 [$1,449] $432 0.004 Pharmacy without ICT $215 [$482] $274 [$544] -$50 0.192 SCD-specific, mean [SD] $2,237 [$3,679] $3,116 [$4,301] -$952 0.0160 Inpatient $1,776 [$3,546] $2,782 [$4,268] -$855 0.0160 Emergency room $18 [$63] $28 [$69] -$199 0.1200 Outpatient $443 [$658] $306 [$548] $105 0.1120 Disclosures Vekeman: Novartis Pharmaceuticals: Research Funding. Sasane:Novartis Pharmaceuticals: Employment. Cheng:Novartis Pharmaceuticals: Research Funding. Ramanakumar:Novartis Pharmaceuticals: Research Funding. Fortier:Novartis Pharmaceuticals: Research Funding. Duh:Novartis Pharmaceuticals: Research Funding. Paley:Novartis Pharma: Employment. Adams-Graves:Novartis Pharmaceuticals: Consultancy, Honoraria, Speakers Bureau.

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