On Location
Conference Coverage
ADVANCES IN TRANSPLANTATION
Attendees in a session at the 2019 TCT Meeting.
t the 2019 Transplantation & Cellular
Therapy Meetings of ASBMT and
CIBMTR, held February 20–24 in
Houston, investigators, clinicians,
researchers, and other health-care
professionals gathered to hear the latest
updates in the field of hematopoietic cell
transplantation and cellular therapy.
Here, we share highlights in hematologic
research presented at the meeting. Look for
more coverage in our May issue.
26
ASH Clinical News
A Safer Conditioning
Regimen for Patients With
Bone Marrow Failure and
Short Telomeres?
A study of patients with bone marrow
failure (BMF) and short telomeres found
that a conditioning regimen that eli-
minates radiation and DNA-alkylating
agents led to successful engraftment and
could spare patients from the toxicities
associated with standard conditioning
regimens.
Removing the “necessary evils” of
DNA-alkylating agents and radiation
could enable transplant in patients with
high-risk comorbidities, according to
lead author Suneet Agarwal, MD, PhD,
from Dana-Farber/Boston Children’s
Cancer & Blood Disorders Center and
Harvard Medical School, who presented
the results as a late-breaking abstract
at the 2019 Transplantation & Cellular
Therapy Meeting.
“Radiation and/or DNA-alkylating
agents have been used to achieve myeloid
engraftment since the inception of allo-
geneic hematopoietic cell transplantation
(HCT),” Dr. Agarwal told ASH Clinical
News. If these preparative regimens can
be avoided, he said, so can the potential
toxicities that accompany them.
For this study, researchers hypoth-
esized that in patients with BMF and short
telomeres, hematopoietic and immune
cells have a replicative disadvantage that
facilitates engraftment of healthy donor
cells without the need for DNA-alkylating
agents and radiation, he explained.
Investigators enrolled 20 patients
with BMF (age range = 1.7-31.5 years at
time of HCT) who were treated at one of
seven institutions between August 2012
and October 2018. All participants had
dyskeratosis congenita (DC) or lym-
phocyte telomere length below the first
percentile, determined by flow cytometry
fluorescent in situ hybridization. The
pre-HCT preparative regimen consisted
of fludarabine and alemtuzumab, and
patients received cyclosporine A and my-
cophenolate mofetil as graft-versus-host
disease (GVHD) prophylaxis.
The primary endpoint of the study was
donor myeloid engraftment, defined as an
absolute neutrophil count ≥500 cells/µL
by 42 days post-HCT and donor chime-
rism greater than 50 percent by 100 days
post-HCT.
April 2019