Breaking News
March 23, 2019 - Excess hormones could cause a condition that can lead to blindness in women, study finds
March 23, 2019 - Dramatic shifts in first-time opioid prescriptions bring hope, concern
March 23, 2019 - Antidepressant drugs may not work when neurons are out of shape
March 23, 2019 - TTUHSC El Paso to establish endowed chair in neurology through a major grant
March 23, 2019 - New device approved by FDA for treating patients with moderate-to-severe heart failure
March 23, 2019 - People with peripheral artery disease have lower Omega-3 Index, shows research
March 23, 2019 - Trigger warnings have minimal impact on how people respond to content, shows research
March 23, 2019 - Gilead Announces Data From Two Studies Supporting Further Development of GS-6207, a Novel, Investigational HIV-1 Capsid Inhibitor as a Component of Future Long-Acting HIV Therapies
March 23, 2019 - Selfish genetic elements amplify inflammation and age-related diseases
March 23, 2019 - Study provides new understanding of how the brain recovers from damage caused by stroke
March 23, 2019 - CRISPR/Cas libraries could revolutionize drug discovery
March 23, 2019 - Allergic reaction during pregnancy may alter sexual-development in offspring’s brain
March 23, 2019 - Seeing through a robot’s eyes helps those with profound motor impairments
March 23, 2019 - Recent research shows that ease of breastfeeding after C-section differs culturally
March 23, 2019 - Newly discovered parameters offer more control over efficient release of drugs
March 23, 2019 - ‘De-tabooing’ of abortion- Women would like more support from health care community
March 23, 2019 - Anti-TB drugs can increase susceptibility to Mtb reinfection
March 23, 2019 - New survey indicates need of attention to neglected tropical diseases
March 23, 2019 - Innovative in vitro method to develop easy-to-swallow medicine for children and older people
March 23, 2019 - Sugary drinks could raise risk of early deaths finds study
March 23, 2019 - Lian wins ENGINE grant for stem-cell-based therapy to treat Type 1 diabetes
March 23, 2019 - Overall, Physicians Are Happy and Enjoy Their Lives
March 23, 2019 - Researchers discover how blood vessels protect the brain during inflammation
March 23, 2019 - CDC study shows modest improvement in optimal hospital breastfeeding policy
March 23, 2019 - Family-based prevention program to reduce alcohol use among older teens
March 23, 2019 - Remote monitoring of implanted defibrillators in heart failure patients prevents hospitalizations
March 23, 2019 - Appropriate doffing of personal protective equipment may reduce healthcare worker contamination
March 23, 2019 - Window screens can suppress mosquito populations, reduce malaria in Tanzania
March 23, 2019 - Researchers discover new biomarker for postoperative liver dysfunction
March 23, 2019 - Pregnancy history may be linked to cognitive function in older women, finds study
March 23, 2019 - Study shows ticagrelor is equally safe and effective as clopidogrel after heart attack
March 23, 2019 - FDA Approves First Drug for Postpartum Depression, Zulresso (brexanolone)
March 23, 2019 - New guidelines outline new treatment management for psoriasis
March 23, 2019 - Thermally abused cooking oil may promote progression of breast cancer
March 23, 2019 - High-fructose corn syrup fuels growth of colon tumors in mice
March 23, 2019 - Partnership aims at establishing best practices to promote diversity in clinical trials
March 23, 2019 - New study examines presence of microbes in tap water from residences, office buildings
March 23, 2019 - Early life trauma may affect brain structure, contribute to major depressive disorder
March 23, 2019 - NIH starts clinical trial of drug to treat cravings associated with opioid use disorder
March 23, 2019 - Cervix bacteria, immune factors could be a warning signal of premature birth, reports new research
March 23, 2019 - Worst-ever emergency care performance figures underscore the need to focus on staffing
March 23, 2019 - The Current issue of “The view from here” is concerned with Cancer
March 23, 2019 - Mouse model validates how ‘good’ and ‘bad’ bacteria affect acne
March 23, 2019 - Individual amygdala neurons respond to touch, imagery and sounds
March 23, 2019 - Combination of two topical creams can prevent cancer
March 23, 2019 - Study suggests depression screening when assessing African-Americans for schizophrenia
March 23, 2019 - New electronic support system for choosing drug treatment based on patient’s genotype
March 23, 2019 - First-of-its-kind study provides pregnancy statistics of imprisoned U.S. women
March 23, 2019 - Marinus Pharmaceuticals Initiates Phase 3 Study in Children with PCDH19-Related Epilepsy
March 23, 2019 - Laparoscopy: MedlinePlus Lab Test Information
March 23, 2019 - Shellfish allergies: can they be treated?
March 23, 2019 - Toilet seat heart monitoring system
March 23, 2019 - Researchers identify way to improve common treatment for PTSD
March 23, 2019 - High potency cannabis use linked to psychosis finds study
March 23, 2019 - Evoke Pharma Submits Response to FDA Review Letter for Gimoti NDA
March 23, 2019 - Tracking HIV’s ever-evolving genome in effort to prioritize public health resources
March 23, 2019 - Scientists grow most sophisticated brain organoid to date
March 23, 2019 - ADHD drug raising risk of psychosis
March 22, 2019 - FDA approves brexanolone, first drug developed to treat postpartum depression
March 22, 2019 - Gruesome cat and dog experiments by the USDA exposed
March 22, 2019 - Ball pits used in children’s physical therapy may contribute to germ transmission
March 22, 2019 - Long-term use of inexpensive weight-loss drug may be safe and effective
March 22, 2019 - FDA Approves Sunosi (solriamfetol) for Excessive Daytime Sleepiness Associated with Narcolepsy or Obstructive Sleep Apnea
March 22, 2019 - Anti-Müllerian Hormone Test: MedlinePlus Lab Test Information
March 22, 2019 - Finding the right exercise, diet aids for HIV patients
March 22, 2019 - Health Plans For State Employees Use Medicare’s Hammer On Hospital Bills
March 22, 2019 - Researchers develop new tool for imaging large groups of neurons in living animals
March 22, 2019 - Certain bacteria and immune factors in vagina may cause or protect against preterm birth
March 22, 2019 - Research identifies guidelines for prioritizing hepatitis C treatment in U.S. prisons
March 22, 2019 - Novel breath test could pave new way to non-invasively measure gut health
March 22, 2019 - Pharmaceutical and personal care products may result in new contaminants in waterways
March 22, 2019 - New model could revolutionize the way researchers investigate spread of pathogens
March 22, 2019 - MSU professor receives NSF CAREER grant for biosensor diagnostics
March 22, 2019 - High-fat, high-sugar diet in mouse mothers causes problems in the hearts of offspring
March 22, 2019 - ACC: Catheter Ablation Does Not Cut Mortality, Stroke in A-Fib
March 22, 2019 - Wiedemann-Rautenstrauch syndrome – Genetics Home Reference
March 22, 2019 - Health insurance is not assurance of healthcare
March 22, 2019 - Supporting “curiosity-driven research” at the Discovery Innovation Awards
March 22, 2019 - Must-Reads Of The Week (Some Flying Below The Radar)
March 22, 2019 - Newly engineered nanoscale protein micelles can be tracked by MRI
Deadly inherited blood disorders cured with bone marrow transplant

Deadly inherited blood disorders cured with bone marrow transplant

image_pdfDownload PDFimage_print

Doubling the low amount of total body radiation delivered to patients undergoing bone marrow transplants with donor cells that are only “half-matched” increased the rate of engraftment from only about 50 percent to nearly 100 percent, according to a new study by Johns Hopkins researchers. The findings, published online Mar. 13 in The Lancet Haematology, could offer a significantly higher chance of a cure for patients with severe and deadly inherited blood disorders including sickle cell anemia and beta thalassemia.

“These results are really exciting as we’re approaching a 90 percent cure rate for sickle cell and beta thalassemia,” says Robert Brodsky, M.D., professor of medicine and oncology research at the Johns Hopkins University School of Medicine, director of the Division of Hematology, and a member of the Johns Hopkins Kimmel Cancer Center. “Bone marrow transplants are not just for patients with a perfectly matched donor. A half-match is definitely good enough.”

In the late 1980s, explain study leaders Javier Bolaños-Meade, M.D., associate professor of oncology at the Johns Hopkins Kimmel Cancer Center, and Brodsky, researchers discovered that bone marrow transplants could potentially cure sickle cell disease, a condition with few effective treatments and one that typically kills patients in their 40s. However, this treatment has only been used sparingly since then. Until recently, it required bone marrow donors and recipients to fully match each other in a set of proteins known as human leukocyte antigens that are displayed on cells. Without a complete match, Brodsky says, the recipient’s body recognizes donor cells as foreign and launches a destructive attack.

Since finding a full match is difficult in this patient population–fewer than 15 percent have fully matched siblings free of the same genetic defect that causes sickle cell disease, and less than a quarter have full matches in unrelated registries–Johns Hopkins researchers developed a protocol, published in 2012, that allows patients to receive transplants from relatives who are only half-matched. This advance significantly expanded the pool of potential donors, Brodsky says, but the resulting transplants only engrafted to produce healthy new blood about 50 percent of the time.

Seeking to increase the odds of engraftment for these half-matches, Bolaños-Meade and his colleagues tested a new protocol for bone marrow transplants in patients with severe sickle cell disease and beta thalassemia, two related blood disorders known as hemoglobinopathies that are caused by defects in the same beta-globin gene. They recruited 17 patients for the study: 12 with sickle cell disease and five with beta thalassemia, with a median age of 16. Each of these patients had a relative who could serve as a half-match for a bone marrow transplant–siblings, mothers, fathers and one aunt.

As in the previous protocol, all received doses of chemotherapy and total body irradiation to knock down their immune response to the donor bone marrow before transplant. Rather than receiving 200 centigray (cGy), the low dose of radiation delivered in the previous protocol, patients in the new study received 400 cGy–still a relatively small amount of radiation that was well-tolerated. After their transplants, all received a dose of cyclophosphamide, a drug that has proved critical to avoiding a potentially deadly condition known as graft versus host disease that is particularly prevalent with half-matches.

Over the next 30, 60, 180 and 360 days, and yearly after that, the researchers tested the patients’ blood for chimerism, the amount of DNA present from their donors that signals that a successful engraftment has taken place. They found that all the patients, except one with sickle cell disease, had successfully engrafted–a rate significantly higher than that seen with the previous protocol. Although five developed graft versus host disease, the condition resolved in each of these patients.

At the time of the study’s publication, only three patients still needed to take immunosuppressive medications. All of those with successful engraftment had either extreme reduction or no symptoms of their disease–the sickle cell disease patients no longer had the pain crises that are hallmarks of their condition. Similarly, the beta thalassemia patients were no longer dependent on blood transfusions.

“These latest findings add to an extensive and growing body of evidence supporting the safety and effectiveness of half-matched bone marrow transplants,” says Richard Jones, M.D., director of the Johns Hopkins Kimmel Cancer Center bone marrow transplantation program. “We have performed more than 1,000 half-matched bone marrow transplants, and our clinical studies have proved so successful, with safety and toxicity comparable to matched transplants, that half-identical transplants must be made available to more patients as a curative option.”

Only mild, low-dose (or “mini”) therapy is needed to allow the transplant to take, making the transplant potentially safer for patients with sickle cell disease and thalassemia who are often unable to tolerate the high-dose (myeloablative) therapy needed for gene therapies to take, Jones says.

Source:

https://www.hopkinsmedicine.org/news/newsroom/news-releases/blood-diseases-cured-with-bone-marrow-transplant

Tagged with:

About author

Related Articles