Archive for research
Most parents of children with advanced cancer don’t recognize their low chance of cure
End-of-life care for children with advanced cancer is often very intensive, and can cause much suffering. When parents recognize that a cure is unlikely, they often choose to spare their children from aggressive treatments and focus on the quality of their child’s remaining time. But a study published Oct. 4 in the journal Cancer finds ... Read More about Most parents of children with advanced cancer don’t recognize their low chance of cure
How families are reshaping Shwachman-Diamond syndrome research
No one knew the heartache about to unfold when Savannah and Brett Lillywhite first began thinking about having a family 10 years ago. The Lillywhites Savannah and Brett are both the unlikely carriers of a rare condition called Shwachman-Diamond syndrome — SDS for short — a blood disorder that can lead to bone marrow failure ... Read More about How families are reshaping Shwachman-Diamond syndrome research
Racial differences in response to asthma therapies, and other AsthmaNet lessons
African Americans have higher rates of serious asthma attacks, hospitalizations, and asthma-related deaths than whites. Now, a large multicenter study of African Americans with poorly controlled asthma finds that one size doesn’t fit all when it comes to common asthma treatments. Results appear in The New England Journal of Medicine (NEJM). The randomized study had ... Read More about Racial differences in response to asthma therapies, and other AsthmaNet lessons
Against all odds: Mila’s unique mutation, and her own custom drug
Ed. note: Mila passed away in February 2021, at age 10. The Mila’s Miracle Foundation continues to work to pave a pathway for personalized treatments. The FDA recently released a draft guidance on testing custom drugs such as Mila’s in patients. As a baby and toddler, Mila was healthy, active, and — in some ways ... Read More about Against all odds: Mila’s unique mutation, and her own custom drug
Meet Meghan: Making clinical research meaningful
I’ve been working for the Experimental Therapeutics and Interventional Trials Center for nearly four years, the last two as a clinical research specialist, serving as a project manager for clinical research studies. What makes my job interesting is doing something a little different every day — anything from startup procedures to coordinating study visits with ... Read More about Meet Meghan: Making clinical research meaningful
Not all heroes wear capes: Taking on dyskeratosis congenita
When Mason Langlais argues with his sister, Jillian, he regularly repeats a mantra. “I’m the rare one,” he says. “So, you can’t treat me like this.” “Rare” and “one in a million” — those are just a few of the words his mom, Jenn, uses to describe her 7-year-old self-proclaimed super hero. It’s how she ... Read More about Not all heroes wear capes: Taking on dyskeratosis congenita
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 about Following clinical trial, boy with Fanconi anemia transfusion free
In surgery for people with sepsis, choice of anesthetic may matter
Sepsis, an extreme immune response to infection, has no specific treatment and is a leading cause of hospital deaths. As part of their care, patients often undergo imaging procedures and surgery to pinpoint and help eliminate the infection. New preclinical findings suggest that the choice of general anesthetic used for these procedures can influence sepsis ... Read More about In surgery for people with sepsis, choice of anesthetic may matter
Pioneering microbiome findings shed light on aspiration
When children have respiratory infections, clinicians tend to blame gastroesophageal reflux, based on the assumption that bacteria-laden stomach contents rise into the mouth and are then aspirated. As a result, clinicians often recommend a type of anti-reflux surgery called fundoplication to treat these infections. Yet, despite undergoing this procedure, many patients don’t improve. A new ... Read More about Pioneering microbiome findings shed light on aspiration
Exploring targeted treatments for children with low-grade brain tumors
Children diagnosed with low-grade astrocytomas, the most common type of pediatric brain tumor, have close to 90 percent overall survival rates. However, the growth of the tumors, as well as the standard treatments (neurosurgery and chemotherapy) required to control them, can cause serious side effects, including damaging developing brains and bodies. Researchers at Dana-Farber/Boston Children’s Cancer and Blood ... Read More about Exploring targeted treatments for children with low-grade brain tumors