Multicenter COMPACT study of COMplications in Patients with sickle cell disease And utilization of iron Chelation Therapy

Abstract

Background:

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.

Objective:

We collected longitudinal data on sickle cell disease (SCD) complication rates and associated resource utilization relative to blood transfusion patterns and iron chelation therapy (ICT) use in patients aged ≥16 years to address a gap in the literature.

Research design and methods:

Medical records of 254 SCD patients ≥16 years were retrospectively reviewed at three US tertiary care centers.

Main outcome measures:

We classified patients into cohorts based on cumulative units of blood transfused and ICT history: <15 units, no ICT (Cohort 1 [C1]), ≥15 units, no ICT (Cohort 2 [C2]), and ≥15 units with ICT (Cohort 3 [C3]). We report SCD complication rates per patient per year; cohort comparisons use rate ratios (RRs).

Results:

Cohorts had 69 (C1), 91 (C2), and 94 (C3) patients. Pain led to most hospitalizations (76%) and emergency department (ED) (82%) visits. 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 ED visits and hospitalizations associated with SCD complications (C2 vs. C3, ED: 1.94 [1.70–2.21]; hospitalizations: 1.61 [1.45–1.78]), but not in outpatient visits.

Conclusions:

Although the most commonly reported SCD complication among all patients was pain, patients who received ICT were less likely to experience pain and other complications than those who did not. These results highlight the need for increased patient and provider education on the importance of comprehensive disease management.

Keywords: :

• Blood transfusions
• Complications
• Iron chelation therapy
• Iron overload
• Sickle cell disease

Transparency

Declaration of funding

This study was funded by Novartis Pharmaceuticals Corporation.

Declaration of financial/other relationships

L.J. has disclosed that she was a paid consultant to Novartis Pharmaceuticals Corporation, and has been a member of the Novartis Speaker Bureau. P.A.-G. has disclosed that she is an employee of the University of Tennessee, Memphis, which has received research funds from Analysis Group Inc. J.K.-W. has disclosed that she was an employee of Tulane University, which has received research funds from Analysis Group Inc. P.A.O. has disclosed that she is an employee of Howard University, which has received research funds from Analysis Group Inc. She has also received honoraria from Novartis Pharmaceuticals Corporation. F.V., C.B., and M.S.D. have disclosed that they are employees of Analysis Group Inc., which has received research grants from Novartis Pharmaceuticals Corporation. M.S., A.M. and M.M. have disclosed that they are employees of Novartis Pharmaceuticals Corporation.

CMRO peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Acknowledgments

The authors would like to thank Zachary Gorn for his assistance in the data collection and data analyses underlying this manuscript. Zachary Gorn was an employee of Analysis Group Inc., which has received research grants from Novartis Pharmaceuticals Corporation.

Previous presentations: The underlying work for this manuscript was previously accepted for poster presentations at the 54th Annual Meeting and Exposition of the American Society of Hematology, Atlanta, Georgia, 8–11 December 2012, Abstract 2106; the 18th Congress of the European Hematology Association, Stockholm, Sweden, 13–16 June 2013, Abstract code P409; the 25th Annual Meeting & Expo of the Academy of Managed Care Pharmacy, San Diego, CA, USA, 3–5 April 2013; and a podium presentation at the 2013 International Conference on Pharmacoepidemiology and Therapeutic Risk Management, Montréal, Canada, 25–28 August 2013, Abstract 964.