SUCCESSOR: A MULTICENTER RETROSPECTIVE NONINTERVENTIONAL FOLLOW-UP STUDY IN PATIENTS WITH SICKLE CELL PAIN CRISES WHO PREVIOUSLY PARTICIPATED IN THE SUSTAIN TRIAL IN THE UNITED STATES
Author(s): ,
Darla Liles
Affiliations:
East Carolina University,Greenville, NC,United States
,
Nirmish Shah
Affiliations:
Duke University Health System,Durham, NC,United States
,
Brigid Scullin
Affiliations:
University of North Carolina Health Care,Chapel Hill, NC,United States
,
Victor Gordeuk
Affiliations:
University of Illinois at Chicago,Chicago, IL,United States
,
Wally Smith
Affiliations:
Virginia Commonwealth University,Richmond, VA,United States
,
Julie Kanter
Affiliations:
University of Alabama at Birmingham,Birmingham, AL,United States
,
Maureen Achebe
Affiliations:
Brigham and Women’s Hospital,Boston, MA,United States
,
Ralph Boccia
Affiliations:
Center for Cancer and Blood Disorders,Bethesda, MD,United States
,
Shelley Crary
Affiliations:
University of Arkansas for Medical Sciences,Little Rock, AR,United States
,
Walter Kraft
Affiliations:
Thomas Jefferson University,Philadelphia, PA,United States
,
Natasha Archer
Affiliations:
Dana-Farber/Boston Children’s Cancer and Blood Disorders Center,Boston, MA,United States
,
Vince Cataldo
Affiliations:
Our Lady of the Lake-Mary Bird Perkins Cancer Center,Baton Rouge, LA,United States
,
Brandon Hardesty
Affiliations:
Indiana Hemophilia and Thrombosis Center,Indianapolis, IN,United States
,
Modupe Idowu
Affiliations:
University of Texas Health Science Center at Houston,Houston, TX,United States
,
Payal Desai
Affiliations:
The Ohio State University Wexner Medical Center,Columbus, OH,United States
,
Alan Ikeda
Affiliations:
Children’s Specialty Center of Nevada,Las Vegas, NV,United States
,
Geetha Puthenveetil
Affiliations:
Children’s Hospital of Orange County,Orange, CA,United States
,
Kathryn Hassell
Affiliations:
University of Colorado Denver School of Medicine,Denver, CO,United States
,
Sharada Sarnaik
Affiliations:
Children’s Hospital of Michigan,Detroit, MI,United States
,
Jincy Paulose
Affiliations:
Novartis Pharmaceuticals Corporation,East Hanover, NJ,United States
,
Dramane Lainé
Affiliations:
Novartis Pharmaceuticals Corporation,East Hanover, NJ,United States
,
Das Purkayastha
Affiliations:
Novartis Pharmaceuticals Corporation,East Hanover, NJ,United States
,
Savita Nandal
Affiliations:
Novartis Pharmaceuticals Corporation,East Hanover, NJ,United States
Abdullah Kutlar
Affiliations:
Sickle Cell Center, Medical College of Georgia, Augusta University,Augusta, GA,United States
EHA Library. Liles D. Jun 15, 2019; 267436; S853
Darla Liles
Darla Liles
Contributions
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Abstract

Abstract: S853

Type: Oral Presentation

Presentation during EHA24: On Saturday, June 15, 2019 from 11:45 - 12:00

Location: Amtrium

Background
SUCCESSOR (SUSTAIN Chart-review of Crizanlizumab to Evaluate Sickle-cell Study One-year Retrospective) reviewed medical records of patients who completed the SUSTAIN study at US sites to assess subsequent cases of significant pain crisis events and generate real-world data on treatment patterns and health care resource utilization upon completion of crizanlizumab treatment. SUSTAIN was a phase 2, randomized, double-blind, placebo-controlled, 52-week study that compared the effect of crizanlizumab, a P-selectin inhibitor, versus placebo on the frequency of sickle cell pain crises (SCPCs, or vaso-occlusive crises [VOCs] leading to a health care visit) in patients with any genotype of sickle cell disease (SCD).

Aims
To determine the 52-week post-SUSTAIN occurrence of VOCs, time to first VOC, frequency of blood transfusions, and use of opioids and hydroxyurea in patients after withdrawal of treatment with crizanlizumab or placebo.

Methods
SUCCESSOR is a multicenter, retrospective cohort study of patients (≥18 years old) who participated in SUSTAIN to evaluate SCD-related outcomes up to 52 weeks following trial completion. SUCCESSOR included the SUSTAIN per-protocol population who received at least 12 of the 14 study drug doses, completed a visit at least 14 days after the final dose, and had no major protocol deviations that impacted efficacy assessments. The study period was from September 2015 to March 2017 and patients’ data were obtained from medical records. Crizanlizumab was not administered in the 52 weeks post-SUSTAIN. An exploratory analysis was conducted to evaluate outcomes in the retrospective SUCCESSOR study using a subset of SUSTAIN patients. Patient consent was obtained prior to data collection if required by local and/or central research ethics review.

Results
In SUCCESSOR, data for 48 patients were analyzed (15, 18, and 15 patients who were previously randomized in SUSTAIN to receive crizanlizumab 5 mg/kg, crizanlizumab 2.5 mg/kg, and placebo, respectively). The median age was 31.5 years (range 19-65 years) and 69% were female. One patient had HbSβ+, 4 had HbSβ0, 10 had HbSC, 1 had HbS-Lepore disease; all remaining patients had HbSS. In the 52 weeks post-SUSTAIN, VOCs were reported by 73% of patients who previously received crizanlizumab 5 mg/kg, 83% of patients who previously received crizanlizumab 2.5 mg/kg, and 80% of patients previously treated with placebo. The mean annual rate of VOCs was 2.7, 3.9, and 5.4 in patients previously on crizanlizumab 5 mg/kg, 2.5 mg/kg, and placebo, respectively. Median time to first VOC post-SUSTAIN was 6.1, 2.7, and 2.6 months in patients previously on crizanlizumab 5 mg/kg, 2.5 mg/kg, and placebo, respectively, similar to differences observed for the same patients during SUSTAIN (6.9, 2.2, and 1.4 months for crizanlizumab 5 mg/kg, 2.5 mg/kg, and placebo, respectively). In SUCCESSOR, all but 1 patient utilized opioids, and 79% did not require blood transfusions. Hydroxyurea was used by 60%, 50%, and 80% of patients previously on crizanlizumab 5 mg/kg, 2.5 mg/kg, and placebo, respectively. There were no deaths.

Conclusion
In SUCCESSOR across all patient groups, 80% of patients experienced a VOC in the 52 weeks post-SUSTAIN. This retrospective cohort study indicates that after withdrawal of crizanlizumab, patients previously treated with 5 mg/kg maintained a similar time to first VOC compared with time to first VOC while on treatment in SUSTAIN. Further research is needed to establish whether these findings are due to a prolonged effect of crizanlizumab or to other factors.

Session topic: 26. Sickle cell disease

Keyword(s): Sickle cell disease, Vasoocclusive crisis

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