Clinical trial NCT06971185: A Study to Evaluate Treatment Patterns and Effectiveness of Luspatercept
Official title: Treatment Patterns and Effectiveness of Luspatercept in the Real World
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Myelodysplastic Syndromes (MDS)
Keywords: Myelodysplastic syndromes (MDS)
Lead sponsor: Bristol-Myers Squibb
Sponsor class: INDUSTRY
First posted: 2025-05-14
Enrollment: 430
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study population will include adult patients identified from the Flatiron Health electronic health record database between January 1, 2011 and August 31, 2024 that have been diagnosed with myelodysplastic syndromes receiving either (a) first-line (1L) luspatercept, (b) 1L erythropoiesis stimulating agents, or (c) second-line luspatercept treatment.

Brief summary: The purpose of this study is to understand the treatment patterns and clinical outcomes of myelodysplastic syndromes patients treated with luspatercept or erythropoiesis-stimulating agents


Eligibility criteria: Inclusion Criteria:

* Included in the Flatiron Health Broad Research Network, with 2 or more visits after January 1, 2011
* Has evidence of diagnosis with myelodysplastic syndromes (MDS) after Jan 1, 2020, as identified by a natural language processing (NLP)-based machine-learning (ML) model
* Has evidence of diagnosis with MDS as identified via structured International Classification of Diseases (ICD) codes:

  * International Classification of Diseases, Tenth Revision, Clinical Modification (ICD-10-CM): D46.x
  * International Classification of Diseases, Ninth Revision, Clinical Modification (ICD-9-CM): 238.7x
* Age ≥ 18 years at MDS diagnosis
* Has either ring sideroblasts positive or negative status, as confirmed by bone marrow aspirate lab results or clinician notes
* Has at least one confirmed structured activity more than 8 weeks prior to the index date

Cohort specific inclusion criteria:

First-line (1L) luspatercept cohort

* Has evidence of receipt of luspatercept as identified via structured data as evidenced by non-cancelled Medication Order or Medication Administration and confirmed via unstructured data
* Has evidence of treatment with luspatercept for at least 12 weeks as evidenced by non-cancelled Medication Orders or Medication Administrations

  1L erythropoiesis stimulating agents (ESA) cohort:
* Has evidence of receipt of any ESA (i.e., epoetin alfa, darbepoetin alfa, epoetin beta, epoetin alfa-epbx, epoetin zeta, or epoetin beta-methoxy polyethylene glycol) for at least 12 weeks as evidenced by non-cancelled Medication Orders or Medication Administrations
* Note: this criterion is included to maximize alignment between the 1L ESA cohort and the 1L luspatercept cohort and minimize bias induced by the dosage requirement in the 1L luspatercept cohort

Second-lin (2L) luspatercept cohort:

* Has evidence of receipt of luspatercept as identified via structured data as evidenced by non-cancelled Medication Order or Medication Administration and confirmed via unstructured data
* Has evidence of receipt of at least 1 ESA as evidenced by a non-cancelled medication order or medication administration prior to the date of initial luspatercept receipt
* Has evidence of treatment with luspatercept for at least 12 weeks as evidenced by non-cancelled Medication Orders or Medication Administrations

Exclusion Criteria:

* Lacking relevant unstructured documents in the Flatiron database for review by the abstraction team
* Have been exposed to any of the following MDS-related therapy prior to luspatercept initiation in the 1L and 2L settings or ESA initiation in the 1L setting: lenalidomide, azacitidine, decitabine, cedazuridine, eltrombopag, cytarabine, daunorubicin, idarubicin, filgrastim, pegfilgrastim, lipefilgrastim, sargramostim, venetoclax, or has evidence of a stem cell transplant

Primary outcomes:
measure: Participant baseline demographics
timeFrame: Baseline
measure: Luspatercept dose at treatment initiation
timeFrame: Baseline
measure: Luspatercept dose at treatment discontinuation
timeFrame: Up to 50 months
measure: Luspatercept dose change or escalation or reduction
timeFrame: Up to 50 months
measure: Time from luspatercept initiation to the first occurrence of dose escalation
timeFrame: Up to 50 months
measure: Proportion of participants that discontinued treatment
timeFrame: Up to 50 months
measure: Time from treatment initiation to treatment discontinuation
timeFrame: Up to 50 months
measure: Time from luspatercept/erythropoiesis stimulating agents treatment initiation to initiation of a new treatment for myelodysplastic syndromes
timeFrame: Up to 50 months
Secondary outcomes:
Other outcomes:
Locations:
city: Princeton
country: United States
facility: Bristol Myers Squibb
state: New Jersey
Study officials:
affiliation: Bristol-Myers Squibb
name: Bristol Myers Squibb
role: STUDY_DIRECTOR
References: