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Blood and Marrow Transplantation Program

at Nicklaus Children's Hospital:

Exceeding Patient Care Standards and Predicted Outcomes

BY DANIEL CASCIATO tion to expand the program by offering
transplant options for other medical
The Blood and Marrow Trans - conditions. Historically, transplants
plantation Program at Nicklaus Chil - were only for kids who had leukemia.
dren's Hospital is South Florida’s first The scope of pediatric diseases treat-
and most experienced care provider for able by stem cell transplant-bone mar-
children requiring blood or bone mar- row transplant now includes, in addi-
row transplantation. tion to malignant disorders such as
As one of only two pediatric blood high-risk acute leukemias and solid
and marrow transplant centers in South tumors, a wide range of hereditary con-
Florida, they perform more than 30 ditions, including storage and metabol-
blood and marrow transplants per year ic disorders, hemoglobinopathies, and
and a total of 400 since its inception in immune deficiencies.”
1991. Bone marrow transplant is now Dr. Maher notes that Florida Blue
commonly referred to as stem cell and several insurance companies have
transplant because they are using other Dr. Jorge Galvez-Silva Dr. Ossama Maher recognized the program with the Blue
sources of stem cells, and not only bone Distinction Center® designation and
marrow, such as cord blood. tation (cellular therapy). The program “Collaboration. We have strong Center of Excellence for transplanta-
“We treat children under the age of continues to meet the rigorous FACT teamwork and collaboration,” he says. tion for delivering high-quality bone
21 years old for several conditions, standards, having achieved its most “The main reason we work well togeth- marrow transplant care. This designa-
such as leukemia and lymphoma and recent reaccreditation in 2020. FACT er is because we put the patients and tion is based on the delivery of quality
other types of tumors and malignan- accreditation is given to organizations their family needs first. Every program specialty care based on objective, trans-
cies,” says Jorge Galvez-Silva, M.D., that exceed standards in patient care puts their patients first, of course. But parent measures for patient safety and
director of the Blood and Marrow and laboratory practices. bone marrow transplant is so complex health outcomes developed with input
Transplant Program at Nicklaus “Another issue that differentiates us that you need to have an open collabo- from the medical community. In addi-
Children’s Hospital, and member of the from other programs is that we are ration with different specialties on your tion, it has been recognized as a center
KIDZ Medical Services team, a physi- located in a fully dedicated, freestand- team, and with the patients and family. of excellence by Blue Cross, Aetna,
cian-owned multispecialty group that ing children's hospital with a multidis- That translates into great outcomes.” Simply, Optum, and Humana.
provides neonatal, pediatric and obstet- ciplinary group of experts, each offer- The key measure of success for any Looking ahead, Dr. Galvez’s hopes
rical healthcare throughout South ing specialties that are really relevant to bone marrow transplant program is the for the future of the field is that they
Florida. The KIDZ team of dedicated the wellbeing of the kids undergoing survival of the patients, according to will be able to cure all children with
pediatric subspecialists, advanced prac- bone marrow transplants,” Dr. Galvez Ossama Maher, M.D., co-director of the cancers.
tice providers, nursing and hearing says. Blood and Marrow Transplant Program “Stem cell therapy is a rapidly evolv-
professionals works in more than a At Nicklaus Children's Hospital, the and also a member of the KIDZ Medical ing field. We’ve come a long way from
dozen hospitals, including Nicklaus KIDZ Medical team provides state-of- Services team. our first attempts to cure various severe
Children’s Hospital. the-art intervention and therapies with “Over the last three years, we’ve been conditions, and as we get more success-
The Blood and Marrow Transplant a level of clinical experience and averaging between 95 to 100 percent ful at it, the community of stem cell
Program’s experience and dedication to patient-focused support that is unparal- survival,” he says. “The national aver- therapists want to achieve survivability
quality and in-depth staff training con- leled in the region. KIDZ Medical is age for a program our size is 75 percent. with less toxicities, so every child can
tinues to drive outcomes that well instrumental in the hematology/oncol- So, our program is doing exceedingly live a normal life,” he adds.
exceed predicted outcomes. It was orig- ogy, pulmonary, and genetics programs well.”
inally accredited in 2003 by the at the hospital. The program has continued to For more information, visit
Foundation for the Accreditation of What makes the program so success- expand over the past few years. www.kidzmedical.com/pediatric-services/
Cellular Therapies (FACT) for all ful can be summed up in one word, “When Dr. Galvez and I joined KIDZ hematology-oncology/.
aspects of blood or marrow transplan- notes Dr. Galvez. Medical, we had the dream and ambi-

Immunotherapy Study Offers Hope for Pancreatic Cancer Patients

In a research letter to the journal JAMA Oncology, treated in a large clinical trial called ASCO TAPUR.
clinical researchers at Sylvester Comprehensive The patient was selected because of their inherited
Cancer Center in the University of Miami Miller BRCA mutation, which led to a relatively high tumor
School of Medicine showed that immunotherapy can mutational burden and other changes that made
be an option for at least one subgroup of pancreatic immunotherapies effective.
cancer patients. The study found that patients with “BRCA and RAD51C mutations make tumors genet-
inherited BRCA and other similar mutations can ically unstable, creating abnormal proteins called
achieve complete responses — in some cases going neoantigens that sensitize the immune system,” said
from near death to durable recoveries. Dr. Hosein. “Pancreas cancer is usually immunologi-
“This is a highly selected subpopulation of patients cally cold — it doesn’t generate an immune response.
with pancreatic cancer who carry specific mutations, But these mutations, which appear in about 5% of
most commonly in their BRCA gene,” said Peter pancreatic cancers, make the tumors immunologically
Hosein, M.D., co-leader of Sylvester’s Gastrointestinal lukewarm and more likely to respond to immunother-
Cancers Site Disease Group, associate professor of apies.”
clinical medicine in the Division of Seeing this success, Dr. Hosein and colleagues
Hematology/Oncology and senior author of the study. Dr. Peter Hosein embarked on their own small study of 12 patients
“Their cancers respond to immunotherapy when with BRCA, RAD51C and similar mutations. The
most pancreatic tumors do not.” drugs were on the market but had not been approved to treat pancreatic cancer, so the
Tumors often evade immune system surveillance by sending false signals, essential- researchers had to use them off-label.
ly telling immune cells they are normal tissue and not cancer. Some immunotherapies All the patients in the study had been treated with chemotherapy and other drugs
thwart this mechanism by blocking these signals and unleashing the immune but had developed resistance. After receiving two different immunotherapies (ipili-
response. While this approach has been effective in several cancers, it has failed often mumab and nivolumab), four patients had complete responses, one had a partial
in pancreatic cancer. response, and two had stable disease — an incredible success rate for patients with
However, in 2017, a single patient with relapsed pancreatic cancer was successfully advanced, treatment-resistant pancreatic cancer.

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10 May 2022 southfloridahospitalnews.com South Florida Hospital News