Brain oxygen supply is different in different regions of the brain of sickle cell disease (SCD) patients who are affected by anemia, which may explain the incidence of silent strokes, a recent study says.

The findings of the study, “White matter has impaired resting oxygen delivery in sickle cell patients,” were published in the American Journal of Hematology.

SCD is a rare genetic disorder caused by mutations in the HBB gene, which provides instructions to make hemoglobin, a protein responsible for blood oxygen transport. When a person carries two faulty copies of HBB, there is poor oxygen blood supply to all tissues and organs in the body. This also includes the brain, an organ for which shortage of oxygen, even for a few minutes, may cause extensive death of neurons.

Emmaus Life Sciences, Inc., a leader in sickle cell disease treatment, announced today that it will provide financial assistance to help eligible patients afford their monthly co-payment for Endari™ (L-glutamine oral powder)1. The program is limited to financially eligible patients covered by commercial insurance.

A significant number of individuals with sickle cell disease are covered by commercial insurance. In some cases, patients may have difficulty affording the monthly co-payment amount.

Mark Diamond, Emmaus' Vice President of Commercialization, commented: "We are committed to removing barriers between patients and Endari – giving a greater number of patients access to our effective treatment for sickle cell disease."

The U.S. Food and Drug Administration (FDA) has awarded fast track status to investigational therapy CTX001 to treat sickle cell disease.

A Phase 1/2 clinical trial (NCT03745287) is recruiting patients.

CTX001 uses the CRISPR gene-editing technology to increase the production of fetal hemoglobin in patients’ red blood cells.

Fetal hemoglobin is a form of hemoglobin that naturally exists when one is born and later is replaced by the adult form of hemoglobin.

For the treatment, doctors collect a patient’s own hematopoietic stem cells (cells from the bone marrow) and modify them in the laboratory so they can produce high levels of fetal hemoglobin.

The U.S. opioid epidemic is not linked to higher in-hospital mortality in patients with sickle cell disease (SCD), according to a national study.

The study came about due to the pronounced increase in opioid-related overdoses and deaths in the U.S. that has been reported since 2000. Its researchers did find increased hospitalization rates in young adult and elderly SCD patients, but not in deaths linked to opioids used to control disease-related pain.

“Opioid Use Is NOT Associated with in-Hospital Mortality Among Patients with Sickle CELL Disease in the United States: Findings from the National Inpatient Sample,” was recently presented at the 60th American Society of Hematology (ASH) Annual Meeting, in San Diego.

Bluebird bio presented new positive results from the ongoing Phase 1/2 HGB-206 clinical trial evaluating the effectiveness and safety of investigational gene therapy LentiGlobin in patients with severe sickle cell anemia.

The company had previously revealed findings from the trial at the end of last year and earlier this year. The new results reflect data collected through September 14.

Results were introduced at the recent 60th Annual Meeting of the American Society of Hematology in San Diego, under the abstract name “Current Results of LentiGlobin Gene Therapy in Patients with Severe Sickle Cell Disease Treated Under Refined Protocol.”

Daily tasks most people don't think too much about, such as eating breakfast, drinking water or even sitting too long, are always top of mind for Jermaine O'Connor.

The 26-year-old has sickle-cell anemia, a genetic blood disorder that can lead to a number of complications, and not taking care of his health can exacerbate his condition, including his chronic pain.

"It can always feel like a very solitary experience," he said. "It actually spills over into sort of everything you like to do or want to do."

O'Connor has had chronic pain since childhood. He'll typically feel it in his hands, arms and legs, but it's hard to predict, and can last from hours to days.

Viewers and people with sickle cell disease have praised TV show Call The Midwife for its storyline portraying the condition.

In Sunday's episode, which is set in 1964, characters Dr Turner and Nurse Trixie Franklin discover that a mysterious disease affecting a young Ghanaian family was a genetic blood disorder.

But it didn't take long for viewers on social media to spot the symptoms and diagnose the condition as sickle cell.

Many took to social media to praise the period drama for its portrayal of the disease.

Acute vaso-occlusive pain is associated with the onset of menstruation, and about 25% of the women with sickle cell disease can differentiate these episodes from menstrual-cramp pain, a study found.

Findings in the study, “Acute Vaso-Occlusive Pain is Temporally Associated with the Onset of Menstruation in Women with Sickle Cell Disease,” published in the Journal of Women’s Health, supported “our clinical experience,” its authors concluded.

Vaso-occlusive crisis (VOC) is a hallmark feature of sickle cell disease (SCD), which is characterized by chronic inflammation, and linked to severe pain due to blocked blood vessels. VOCs are the most common cause of emergency room visits and hospitalizations in sickle cell patients. They can severely affect a patient’s quality of life, and sometimes lead to death.

Emmanuel “Manny” Johnson, 21, of Brockton was the first patient to participate in a novel sickle cell clinical trial at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center — taking the risk for the sake of his little brother, who has the same disease.

Staying at the hospital for five or six hours every month for a blood transfusion was something that Johnson described as “a regular thing.” He had been receiving transfusions since he was 4-years-old after sickle cell caused him to have two mild strokes.

Now, Johnson hasn’t had a transfusion in nine months, thanks to the clinical trial. The trial wasn’t easy, but one thing motivated Johnson to stay strong — his 7-year-old brother, Aiden, who also has sickle cell disease.

Doubling the dose of total-body irradiation (TBI) led to a near-doubling of the engraftment rate for patients with severe hemoglobinopathies undergoing haploidentical transplants, a small clinical trial showed.

All but one of 17 patients had successful engraftment after receiving 400 cGy TBI as part of a nonmyeloablative conditioning regimen. Following successful engraftment, 15 of 16 patients remained transfusion independent, and 14 of 16 discontinued immunosuppression.

Four patients developed grade 2 acute graft-versus-host disease (GVHD) and one developed grade 3, but GVHD had resolved in all cases at the most recent follow-up, Javier Bolaños-Meade, MD, of Johns Hopkins Sidney Kimmel Comprehensive Cancer Center in Baltimore, and co-authors reported online in The Lancet Haematology.