Aplastic anemia is a rare but serious blood disorder that occurs when the body’s bone marrow cannot produce enough healthy blood cells to function properly. Aplastic anemia can occur in both children and adults. Most cases of aplastic anemia are idiopathic, meaning the underlying cause is unknown. The blood disorder can have inherited (genetic) causes … Continued
Blood disorders, childhood
This post was originally published on Vector, Boston Children’s Hospital’s science and clinical innovation blog. Back in the 1950s, doctors began using steroids to treat Diamond-Blackfan anemia, or DBA, a severe condition in which patients cannot make enough red blood cells. There was no real rationale for using steroids, but there was no other good option, … Continued
This post was originally published on Vector, Boston Children Hospital’s science and clinical innovation blog. In 1938, Louis K. Diamond, MD, and Kenneth Blackfan, MD, at Boston Children’s Hospital described a severe congenital anemia that they termed “hypoplastic” (literally, “underdeveloped”) because of the bone marrow’s inability to produce mature, functioning red blood cells. Eighty years … Continued
Emmanuel “Manny” Johnson, Jr., shares many loves with his little brother, Aiden—from basketball to video games. One thing he wishes they did not share is sickle cell disease (SCD), so Manny is playing a role in a new effort to improve treatment for patients like seven-year-old Aiden, himself, and others living with the inherited blood … Continued
In the context of a diagnosis of cancer, anemia can be caused by the cancer itself, chemotherapy treatments, or both.
Eli Morse has had to make major changes to his active lifestyle, but one thing has stayed the same: Eli and his family will continue to fight his aplastic anemia together.
In 2009, Drew was diagnosed with aplastic anemia, a rare blood disorder. Because of a stem cell transplant, he was cured—and now, he has his mind set on becoming a pediatric oncologist.
Leukemia is a type of cancer that starts in early forms of white blood cells. White blood cells are cells that typically fight infections.
There are many types of gene therapy, but all of them involve adding to or modifying the genetic program in a person – his or her DNA blueprint that provides instructions for building and operating the body. Gene therapy technology has been developed over several decades and has been successfully used to correct rare blood … Continued
This originally appeared on Vector, Boston Children’s Hospital’s blog. A novel screening method using CRISPR-Cas9 genome editing technology has revealed new drug targets that could potentially enhance the effectiveness of PD-1 checkpoint inhibitors, a promising new class of cancer immunotherapy. The method, developed by a team at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, uses CRISPR-Cas9 … Continued
In their Brookline home-far-away-from-home, 2-year-old Duy Anh “Harry” Le plays with blocks and pop-up toys on the floor with his mother, Thao Nguyen. He is lively and happy, and his skin is clear. He looks almost nothing like the sickly baby covered in eczema who arrived in Boston from his native Vietnam in November 2016 … Continued
Tracy Antonelli was four when she was diagnosed with thalassemia, a rare blood disorder that occurs often enough in Mediterranean countries like Italy that an old adage, uttered only partially in jest, warns Italian-Americans against marrying other Italian-Americans. In 2002, a grown-up Antonelli wed Patrick Mooty, whose background is mostly Irish. Their three daughters – … Continued
In March 2016, Ollie the pug, a therapy dog at Boston Children’s Hospital, paid a bedside visit to 7-year-old Carter Mock. Both dog and boy lost limbs to osteosarcoma, a cancer of the bone. Ollie’s left front leg was amputated at the shoulder. After removing the tumor in Carter’s left leg bone, surgeons fashioned a … Continued
Having a child diagnosed with a life-threatening illness is heart-wrenching for all parents, but when the diagnosis itself is uncertain, parents can face excruciatingly difficult decisions. This is what Katie and Josh Stevens of Idaho confronted after their son Riley was diagnosed in October 2012 with the blood disorder aplastic anemia, in which the body’s … Continued
Some 100 days after receiving a stem cell transplant to cure his severe aplastic anemia, 13-year-old Behaylu Barry still couldn’t invite friends into his home. He can’t return to school until January, when his immune system will finally be strong enough to fight the pathogens present in indoor spaces. Yet Behaylu was doing so well … Continued
Steven Clifford is an 18-year-old osteosarcoma survivor. A Boston native, he starts college at the University of California, San Diego this month. This is his story. Life is made up of many difficult decisions. However, imagine my surprise when I had to make a tough and potentially life changing decision at the young age of … Continued