Archive for blood
Scarlette: Hoping for a bright future with PK deficiency
Two-year-old Scarlette Walker loves pink. For her January visit to the Hematology Clinic at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, she wore a shocking pink hat and pink dress. “She picked them out herself,” says her mom, Olivia. She even has a pink alter ego. “She pretends she is a pink helicopter named Dizzy. If ... Read More
Good early results with gene therapy for rare immune deficiency
Brenden Whittaker, a college student in Ohio, has been caught off guard by his good health. Since he was young, a rare immune deficiency known as chronic granulomatous disease (CGD) had left him vulnerable to life-threatening infections. He was used to going in and out of the hospital, and then hooking up to an IV ... Read More
Bone marrow-on-a-chip provides new research directions for Shwachman-Diamond syndrome
A new research tool that mimics the behavior of diseased bone marrow provides a new strategy for understanding the bone marrow disease, Shwachman-Diamond syndrome (SDS), and hopefully, developing new treatments. With SDS, bone marrow fails to produce blood cells normally, leading to bone marrow failure and an increased risk of leukemia. In a research paper ... Read More
Decoding sickle cell disease offers new outlook for Lamarcus
When Lamarcus Jean visits the Hematology Clinic at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, he makes himself right at home. The 6-year-old, whom his mom, Stephanie, describes as “wise beyond his years,” has been a patient here since he was born. Lamarcus has sickle cell disease, an inherited blood disorder caused by a mutation ... Read More
A potential Diamond-Blackfan anemia treatment swims into view
Zebrafish, besides being popular in aquariums, make good stand-ins for studying human diseases. They share about 70 percent of their genes with humans, and can be studied at a mass scale, enabling scientists to test hundreds, even thousands of drugs at a time simply by adding the drug to their water. One such test came ... Read More
A big step toward curbing graft-vs.-host disease after bone marrow transplant
A drug used for rheumatoid arthritis has moved a step closer to FDA approval for a desperately needed new use. The drug, abatacept, has gained FDA Breakthrough Therapy Designation for preventing acute, severe graft-versus-host disease (GVHD) in patients receiving bone marrow transplants. That could help fast-track the drug to the clinic. “If we are lucky ... Read More
How a broken leg and a transatlantic flight led to a deep vein thrombosis
Pain was 16-year-old Katerina Dalmanieras’s first warning sign when she returned from a family vacation in August. The high school junior, who fractured her fibula a month earlier, was in a leg brace and on crutches, so she had not moved during the seven-hour transatlantic flight. “When I got home from the airport, my leg ... Read More
An education in hemophilia for Colin’s new school
Every morning, one of Colin Bazinsky’s parents puts a needle into his chest to give him an infusion of clotting factor. You wouldn’t guess anything was amiss from the placid look on the 3-year-old’s face as he receives the infusion to treat his hemophilia. His state of calm reflects his parents’ matter-of-fact approach to the ... Read More
Following clinical trial, boy with Fanconi anemia transfusion free
Seven-year-old Ervis of Chicago, Illinois, is a model student with a positive attitude and a megawatt smile. His mom Ofelia calls him “un encantador” — “a charmer,” but life as Ervis knows it is not exactly charmed. Born with Fanconi anemia (FA), a rare hereditary blood disorder that can lead to bone marrow failure and cancer, ... Read More
Breaking records: Bob Watson approaches 850 platelet donations
When 68-year-old Bob Watson first donated platelets in 1985 at the Boston Children’s Hospital Blood Donor Center, he sat in one of only two platelet donation beds with needles in both arms — one to take out the blood, the other to return red blood cells and plasma. At the time, Boston Children’s was a much ... Read More