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Pediatric BMT Specialty Update

September 2015 - Blood and Marrow Transplantation

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— Michael Verneris, MD

Clinical trial of engineered T-cell therapy enrolling

The Pediatric Blood and Marrow Transplantation Center is now enrolling patients in a single arm, open-label, multicenter, phase 2 study to determine the efficacy and safety of experimental chimeric antigen receptor (CAR)-modified T-cell therapy in pediatric patients with relapsed or refractory B-cell acute lymphoblastic leukemia. The local primary investigator is Michael Verneris, MD.

To be eligible, patients must be 2 to 21 years of age and meet one of the following criteria: Experiencing a second or later relapse; any relapse after allogeneic stem cell transplantation (Patient should be at least 6 months post-transplant at the time of enrollment.); lack of complete response after 2 cycles of a standard chemotherapy regimen; patients with Philadelphia chromosome positive (Ph+) ALL are eligible if they are intolerant to or have failed 2 lines of tyrosine kinase inhibitor therapy or if tyrosine kinase therapy is contraindicated or if they are ineligible for allogeneic stem cell transplantation.

Impact of cryopreservation on outcomes

Umbilical cord blood is often collected and cryopreserved for years before use in stem cell therapy, but until now the precise impact of cord blood age on clinical outcomes was unknown. Investigators have recently found that the time spent in cryopreservation has no impact on the recovery of total nucleated cells from a cord blood unit nor on the unit’s post-thaw viability. Perhaps most importantly, the duration of cryopreservation had no effect upon neutrophil engraftment or any other clinical outcome, suggesting that storage of cord blood had no impact on clinical outcomes and that unit age should not be used when choosing a unit for transplantation. The investigators analyzed 288 single cord blood units used for transplantation from 1992 to 2013, with unit cryopreservation time ranging from .08 to 11.07 years. Michael Verneris, MD, Troy Lund, MD, PhD, John E. Wagner, MD, and colleagues published the study results in the January 2015 issue of Biology of Blood and Marrow Transplantation.

Role of natural killer cells in stem cell transplants described

After hematopoietic stem cell transplants, reconstituted natural killer (NK) cells represent the predominant lymphoid cells, playing a key role in preventing viral infections and restraining residual cancer cells. Cytotoxic lymphocytes of the innate immune system, NK cells are capable of infiltrating tissues, where they can trigger apoptosis of malignant cells. Recently, Bone and Marrow Transplantation Center physicians published a comprehensive review of the cancer-control function of NK cells in the transplant setting. The article includes a discussion of current research aimed at improving NK cell responses for therapeutic benefit, such as combining NK cell infusions with the best available stem cell transplant strategies. Michael Verneris, MD, Bruce Blazar, MD, John E. Wagner, MD, and Jeffrey S. Miller, MD, authored the article appearing in the June 3, 2015, issue of Current Topics in Microbiology and Immunology.

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