This study is a prospective, non-randomized feasibility study of drug response profiling (DRP) in pediatric blood cancers. Primary cancer cells are isolated from patients and screened ex vivo at single-cell resolution using automated fluorescence microscopy. Drug sensitivity fingerprints are integrated with genetic annotations to inform the treating physician about personalized treatment options. The study aims to determine the practicability of real-time drug response profiling and its actionability in identifying patient-specific cancer dependencies in refractory disease settings.
Pediatric and adult patients below the age of 40 years
Diagnosis of hematological malignancy (primary, relapsed or refractory) including leukemia, myeloma or lymphoma
Tumor material collected as part of routine diagnostics and willingness to donate tumor material for translational research
Patient and/or guardian has signed the informed consent of the DRP registry or of a clinical trial which includes DRP as add-on research.
Missing informed consent for the registry or of a clinical trial which includes DRP as add-on research
Pediatric and adult patients with hematological malignancies who have undergone bone marrow aspiration, peripheral blood or lymph node biopsies as part of routine diagnostics.
Cohort
Prospective
Not listed
Feasibility of real-time ex vivo drug screening
Rate of successful single-cell, image-based drug response profiling assays which are reported to the treating physician.
Translation to clinic
Percentage of DRP assays translated into the clinic, i.e. the referring physician decides to treat the patient with one or more of the top-ranking drugs identified by DRP
In vitro sensitivity of cancer cells to drug perturbations
Exploring drug sensitivity and resistance patterns by ex vivo DRP and correlations to genetic characteristics and clinical response.
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