56. Jahrestagung der Deutschen Gesellschaft für Transfusionsmedizin und Immunhämatologie

Prof. Dr.

Vorsitz

Alternative curative treatment options for sickle cell disease and transfusion dependent thalassemias

Vortrag

CRISPRing the Genome: Wichtige Lehren aus der Bewertung von On- und Off-Target-Effekten


eingeladene Redner

PD Dr.

Characterization of stem cell product quality in vitro and by in vivo function through hematopoietic engraftment

Allogeneic regenerative Cell Therapy

Background Stem cell transplantation is a quality-controlled key element in treatment of hematological disorders. Characterization of stem cell products follows internationally standardized criteria and is supervised by accreditation standards. We aimed to characterize the quality of all allogenic stem cell products obtained in our collection center produced within a 5-year period, and investigate stem cell function by engraftment criteria in two subcohorts of related donors transplanted in our hospital.Methods A total of 193 G-CSF mobilized peripheral blood (PBSC) and 74 bone marrow (BMSC) products collected 2018-2022 were analyzed for a total of 27 parameters including volume, CD34+ and T cell content. Graft function was analyzed in a cohort of adult patients with malignancies receiving PBSC (n=24) and pediatric mainly nonmalignant patients receiving BMSC (n=12). Successful engraftment was defined as neutrophil (NEU, &gt;0.5 G/l), platelet (PLT &gt; 50 G/l) and complete donor chimerism (&gt;90%) determined by STR analysis and absence of overt relapse. Median follow-up for patients in the subcohort of related transplantations was 321 days.Results Unrelated PBSC contained a median of 7.9 x106 (range: 3.2–28.9) and related PBSC a median of 7.5 (range: 3.4–22.1) x106 CD34+/kg. Unrelated BMSC contained median 3.3 (range: 1–22.2) and related BMSC median 5.7 (range: 1.6–20.7) x106 CD34+/kg. In the related cohorts, median transplanted cell doses were 8.2 PBSC x106 CD34+/kg (3.4–22.1) and 7.0 x106 BMSC CD34+/kg (1.58–12.32). Median time to NEU recovery in days was 17 (10–23; PBSC) and 22 (14–28; BMSC) and to PLT recovery 15 (12–37; PBSC) and 22 (15–37; BMSC). Complete donor chimerism was reached in 20/24 of PBSC and 12/12 of BMSC patients. In the PBSC group, 1 patient died before engraftment and 1 rejected the graft. In BMSC, high graft volumes were difficult to apply in most children. Conclusion Guideline-conform in vitro product quality was reached throughout. Engraftment analysis indicated a slightly slower NEU and PLT engraftment in the BMSC compared with PBSC cohort. All but 2 PBSC patients reached engraftment. Grade 3 and 4 GvHD was so far observed in 1 PBSC and 1 BMSC transplant recipient. Pediatric BMSC could be optimized by volume reduction. Our analysis demonstrates that conform in vitro transplant quality is associated with robust stem cell regenerative function in our center.Offenlegung Interessenkonflikt: keineInvited talks abstract/summary

Freier Vortrag

Dr. Dr.

Allogeneic cryoPBSC, DKMS-1: Banking of the world's first allogeneic undirected cryopreserved hematopoietic stem cell transplant

Biobanking and Tissue Banking

Background Cord blood transplants are a promising opportunity for patients with hematological diseases. However, application rates are declining, complicating the economic sustainability of public cord blood banks. The DKMS Stem Cell Bank has established high standard processes in terms of processing, cryopreservation, quality control, transport and quality management. These represent excellent conditions for the development, processing and storage of further cell products.Methods For the further development of a cord blood bank into a stem cell bank, existing processes, facilities and resources were used to expand activities. An existing quality management system and official approvals were built on. The technical requirements of the cord blood bank were adopted and expanded for the stem cell bank. The manufacturing process had to be implemented and validated, the quality control methods had to be adapted to the new cell product and re-validated. Further adjustments were made, e.g. with regard to the integration of apheresis centers, the expansion of the IT infrastructure, the addition of official approvals and the implementation of the PBSC listing via the DKMS Registry.Results In the new GMP clean room unit of the DKMS Stem Cell Bank, allogeneic undirected cryopreserved PBSC products are produced with 5% DMSO and a volume of 2x100 ml. The quality of each batch is verified using validated quality control methods. The documentation is carried out using GMP-compliant software. The high quality of the stem cell products was verified both before freezing and after thawing and after 18 months of storage as part of approval-relevant validations. In 2022, the manufacturing license of the DKMS Stem Cell Bank was extended to include allogeneic undirected cryopreserved PBSC preparations. In 2023, approval was granted for "Allogeneic cryoPBSC, DKMS-1", the world's first allogeneic undirected cryopreserved PBSC product.Conclusion The DKMS Stem Cell Bank has evolved from a pure cord blood bank to a stem cell bank. With "Allogeneic cryoPBSC, DKMS-1" the world's first allogeneic undirected cryopreserved PBSC product was approved. The advantages of this ready-to-use drug are a significant reduction of the time-to-transplant, the improved possibility of transplantation coordination and the previously known product specifications of the selected preparation.Offenlegung Interessenkonflikt: Keine

MOA 15

Sektion Stammzelltransplantation und Zelltherapie

Sektionssitzungen

Sektion Stammzelltransplantation und Zelltherapie

Termin

Vorsitz

Programm