Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at San Diego, California and other locations
Dates
study started
study ends around
Principal Investigator
by Rafael Bejar
Headshot of Rafael Bejar
Rafael Bejar

Description

Summary

This observational, multi-center study aims to collect data in order to develop a novel, minimally invasive diagnostic tool for MDS based on peripheral blood profiling of circulating hematopoietic stem and progenitor cells (cHSPCs) using single-cell RNA sequencing and DNA sequencing.

Official Title

Development of a Peripheral Blood Assay to Replace BM Evaluation in Cytopenia - a Multi-Center Observational Study

Details

This observational, multi-center study aims to collect data in order to develop a novel, minimally invasive diagnostic tool for MDS based on peripheral blood profiling of circulating hematopoietic stem and progenitor cells (cHSPCs) using single-cell RNA sequencing and DNA sequencing. Current MDS diagnosis relies on bone marrow (BM) aspiration and biopsy, which is invasive, technically challenging, and sometimes inconclusive, particularly in early or low-risk disease. Our prior research established a healthy reference map of cHSPC subtypes and developed a computational pipeline that distinguishes MDS from non-clonal cytopenias based on deviations from this reference. In this study, patients referred, as part of standard care, for BM evaluation due to unexplained cytopenias or abnormal blood counts and undergo PB collection for genomic profiling. The study's primary objective is to develop a PB-based test by comparing the developed test diagnoses to the conventional BM-based one. Secondary objectives include evaluating its potential for MDS risk stratification and assessing its ability to reduce the need for BM evaluations.

Keywords

Cytopenia, MDS, PERIBLOOD-MDS

Eligibility

You can join if…

Open to people ages 18 years and up

  1. Patients aged 18 and up with suspected/confirmed MDS cases referred to BM evaluation either for diagnosis or risk assessment due to cytopenia
  2. Platelets < 150 × 10E9/L or
  3. Absolute neutrophil count < 1.8 × 10E9/L or
  4. Hemoglobin (Hgb) < 13 g/dL (males) and < 12 g/dL (female) and
  5. For both sexes, no evidence of Iron, folinic acid, or B12 deficiency

You CAN'T join if...

  1. Women who are pregnant
  2. Previous diagnosis of leukemia; AML, MPN, ALL, CLL, MGUS/MM or any other gammopathy
  3. Lymphocytes > 5000/ul
  4. Patients who are on disease-related therapy are excluded, unless they are treated with Erythropoietin or Prednisone. See Appendix 2 for the list of excluded treatments.
  5. Patients who have undergone a bone marrow transplant.

Locations

  • University of California San Diego accepting new patients
    San Diego California 92093 United States
  • University of Miami accepting new patients
    Miami Florida 33146 United States

Lead Scientist at UCSD

  • Rafael Bejar
    Our lab is focused on understanding the genetic changes that drive the development and progression of hematologic malignancies like acute myeloid leukemia and myelodysplastic syndromes. Our goal is to translate our discoveries into clinically meaningful improvements in how we care for patients with these disorders.

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Weizmann Institute of Science
ID
NCT07081087
Study Type
Observational
Participants
Expecting 1500 study participants
Last Updated