A Open Label, Post Marketing Surveillance Study Following Transfusion of INTERCEPT Platelet Components

This study is a prospective, non-randomized sequential cohort, open label, multi-center, non-inferiority, Phase IV surveillance study following transfusion of INTERCEPT PCs. The patient population will be hematology-oncology patients, including those undergoing hematopoietic stem cell transplant (HSCT), expected to require one or more PC transfusions. For each participating center, the study will start with a brief pilot run-in period with a group of at least 5 patients exposed only to conventional PCs. The purpose of this pilot run-in is to evaluate study logistics and data collection methods within each study center. Data from the pilot phase will be included in the data analysis for the treatment comparison. After the pilot run-in period, the study will be conducted in two sequential patient cohorts: 1) the Control cohort during which study patients will receive only conventional PCs, and 2) the INTERCEPT cohort during which patients will receive only INTERCEPT PCs. Patient enrollment at each Center will be monitored to target similar numbers of patients into the Control and Test Cohorts within each center. Centers may enroll Control and Test patients in ratios that vary from 2:1 to 1:2 due to institutional requirements to move rapidly to full INTERCEPT implementation, or due to availability issues with either Test or Control components. Within each Center, patient enrollment will be stratified in four categories: (1) chemotherapy only; and by use of conditioning regimens for hematopoietic stem cell transplantation (HSCT) in (2) myeloablative conditioning, (3) non-myeloablative conditioning, and (4) reduced intensity using the Center for International Blood and Marrow Transplant Research (CIBMTR) criteria. Note time from last chemotherapy treatment to first study transfusion should be no more than 30 days. To ensure both Test and Control cohorts have a similar allocation ratio (±10% per category) among the conditioning regimen strata, enrollment caps will be set for the Test cohorts, hence no Test patients will be enrolled to a stratum once the cap for the given stratum is met. Eligible patients will be enrolled in open Test strata sequentially as long as there is sufficient Test PC inventory available. Enrollment may be delayed for the Test cohort if sufficient inventory of Test PCs is not available.

Sex

All

Inclusion Criteria:

* Patients with a hematology-oncology disorder expected to require or requiring a transfusion of one or more PCs (time from last chemotherapy treatment to first study transfusion should be 30 days or less).
* Written signed informed consent (unless exemption of individual consent is granted by the center's IRB).

Exclusion Criteria:

* Assisted ventilation (administered by intubation or tight fitting mask with PEEP or CPAP ≥ 5 cm H2O) within 30 days prior to the first study PC transfusion. In the event that PEEP or CPAP values are unavailable for prior assisted ventilation events administered by intubation or tight fitting mask, they will be interpreted as meeting the exclusion criterion. For the purposes of this study, elective intubation of pediatric patients for the short-term protection of the airway during medical or surgical procedures does not qualify as assisted ventilation, provided that there is no parenchymal pulmonary lesion 24 hours after extubation.
* Documented allergy to psoralens

• DERIVEDWheeler AP, Snyder EL, Refaai M, Cohn CS, Poisson J, Fontaine M, Sehl M, Nooka AK, Uhl L, Spinella PC, Fenelus M, Liles D, Coyle T, Becker J, Jeng M, Gehrie EA, Spencer BR, Young P, Johnson A, O'Brien JJ, Schiller GJ, Roback JD, Malynn E, Jackups R, Avecilla ST, Liu K, Bentow S, Varrone J, Benjamin RJ, Corash LM. Acute pulmonary injury in hematology patients supported with pathogen-reduced and conventional platelet components. Blood Adv. 2024 May 14;8(9):2290-2299. doi: 10.1182/bloodadvances.2023012425. PMID 38447116
• DERIVEDDelaney M, Karam O, Lieberman L, Steffen K, Muszynski JA, Goel R, Bateman ST, Parker RI, Nellis ME, Remy KE; Pediatric Critical Care Transfusion and Anemia EXpertise Initiative-Control/Avoidance of Bleeding (TAXI-CAB), in collaboration with the Pediatric Critical Care Blood Research Network (BloodNet), and the Pediatric Acute Lung Injury and Sepsis Investigators (PALISI) Network. What Laboratory Tests and Physiologic Triggers Should Guide the Decision to Administer a Platelet or Plasma Transfusion in Critically Ill Children and What Product Attributes Are Optimal to Guide Specific Product Selection? From the Transfusion and Anemia EXpertise Initiative-Control/Avoidance of Bleeding. Pediatr Crit Care Med. 2022 Jan 1;23(13 Suppl 1 1S):e1-e13. doi: 10.1097/PCC.0000000000002854. PMID 34989701