Following a report earlier this year that drew attention to the challenges of living with sickle cell disease and the urgent need for change in patient care – particularly in hospital emergency departments – Pfizer Rare Disease brought together the Sickle Cell Disease Council for Change (CFC). The CFC consists of more than 20 national Sickle Cell Disease advocates, patients, and experts who tackle the pressing issues faced by those living with the disease. The CFC is focused on improving quality of patient care along with enhancing education and advocacy for the Sickle Cell Disease community. Sickle Cell Disease is a lifelong debilitating blood disorder that causes reduced blood and oxygen flow in the body, often resulting in organ damage and severe pain.  

  Nigerians are paying tribute to popular radio presenter and entertainer Tosyn Bucknor, who has died aged 37. Bucknor died from complications due to sickle cell, her sister and renowned event planner, Funke Bucknor Obruthe said in an Instagram post. "My darling sister and besto Tosyn passed away last night due to complications from sickle cell," she wrote. Bucknor was found unconscious by her husband, a French national, Aurélien Boyer when he returned home from work on Monday night, according to local media reports. Known as the 'Voice of Lagos,' her early morning shows aired on two local radio stations and attracted a strong fanbase among young Nigerians. She was vocal about her health condition and founded the 'These Genes Project' in 2007 to raise awareness about sickle cell.  

An inexpensive, easy-to-use diagnostic tool for sickle cell disease showed 100% accuracy among a large cohort of children from Uganda, according to findings presented during the late-breaking abstract session of ASH Annual Meeting and Exposition.

The test — HemoTypeSC (Silver Lake Research Corporation) — only requires a drop of blood and produces results in about 10 minutes.

Diagnosis, prophylaxis, counseling and treatment have dramatically reduced sickle cell disease incidence and mortality in the developing world, but screening for the disease has not been widely implemented in those regions, particularly in sub-Saharan Africa, according to Erik Serrao, PhD, director of business and product development at Silver Lake Research Corp.

“Although this disease is acknowledged as a public health problem by the U.N. and WHO, African governments and ministries of health still largely neglect this disease,” he said during a press conference. “Individuals with this disease are commonly identified only after they die or when they present at the hospital with life-threatening symptoms.”

  After over a decade of preclinical research and development, a new gene therapy treatment for sickle cell anemia (SCA) is reversing disease symptoms in two adults and showing early potential for transportability to resource-challenged parts of the world where SCA is most common. Preliminary data from a pilot Phase 1-2 clinical trial testing the gene-addition therapy were presented on Dec. 3 at the American Society of Hematology's (ASH) annual meeting in San Diego by principal investigator Punam Malik, MD. She is a physician-scientist at the Cincinnati Children's Cancer and Blood Diseases Institute and director of its Comprehensive Sickle Cell Center. Malik called the data promising. One year after treatment of our first patient, and six months after treatment of our second patient, both have seen a remarkable improvement in the quality of life due to remarkable reduction in disease symptoms.  

 

Global Blood Therapeutics, Inc. (GBT) announced that the U.S. Food and Drug Administration (FDA) has informed GBT through discussions and written correspondence that the agency agrees with the Company’s proposal relating to use of an accelerated approval pathway for voxelotor for the treatment of sickle cell disease (SCD). GBT plans to submit an NDA for voxelotor for the treatment of SCD under this pathway. GBT proposed that by raising hemoglobin, voxelotor is reasonably likely to reduce strokes in SCD patients. As part of these discussions, the FDA agreed that transcranial doppler (TCD) flow velocity would be an acceptable primary endpoint in a post-approval confirmatory study to demonstrate stroke risk reduction.

  The Sickle Cell Disease Association of America, Inc., the Sickle Cell Foundation of Georgia, Inc. and the American Red Cross announce a new five-year partnership and the launch of national blood drive and educational campaign that will enhance awareness about sickle cell disease while addressing critical blood supply needs. The initiative will also target African-American blood donors and highlight the need for a stable supply of blood donors of African descent. Through collaboration with community-based organizations, advocacy groups, colleges and universities, hospitals, and others, this partnership will help to ensure patients have access to lifesaving blood products. The annual goal of the campaign is to mobilize 100 blood drives (with a minimum of 30 units collected per drive) targeting African-American donors across the nation. Over the five-year partnership, it is anticipated that upwards of 15,000 blood donations will be made, helping to save thousands of lives. "Sickle Cell Disease Association of America is excited about this new partnership and the impact it can have on saving lives," said SCDAA President/CEO Beverley Francis-Gibson.

  The American Society of Hematology (ASH) previewed clinical guidelines on sickle cell disease (SCD) here, with the intention of releasing the full set of guidelines in 2019. The chairs from each of the five SCD guideline panels -- sickle cell disease-related transfusion support; sickle cell disease-related cardiopulmonary and kidney disease; sickle cell disease-related cerebrovascular disease; sickle cell disease-related pain; and stem cell transplantation for sickle cell disease -- discussed a key recommendation from each of their panels at the ASH annual meeting. These five SCD panels, which included more than 75 experts across the field, have formulated more than 50 recommendations on SCD. Some draft recommendations veer away from standard practice, or even address issues that haven't been addressed in previous SCD guidelines. For example, John F. Tisdale, MD, of the National Heart, Lung, and Blood Institute in Bethesda, Maryland, pointed out that ASH is putting out the first set of guidelines addressing transplantation in SCD, and noted the stem cell transplantation panel's recommendation of "transplantation at a younger age over transplantation at an older age."

  Students with sickle cell disease (SCD) feel that educators and administrators at their schools lack knowledge about SCD management — an issue that could be addressed by developing a handbook that staff could use to help students with SCD. The study with that finding, “Student perspectives on managing sickle cell disease at school,” was published in the journal Pediatric Blood & Cancer. Most children with SCD are able to lead normal lives, despite complications, such as acute or chronic pain crisis, which occur as a result of the disease. However, they require specific assistance to maintain their health and prevent complications that can affect them daily. In a home setting, monitoring the needs of a child with SCD tends to be more feasible. On the other hand, during the day, children spend most of their time in school, an environment in which SCD episodes can be challenging.  

  Many sickle cell disease (SCD) patients use marijuana for its medicinal benefits, according to an anonymous survey by researchers from Yale New Haven Hospital and Yale University. These results show that there is a need to study the effects of marijuana on these patients and to consider whether sickle cell disease should be included in the list of conditions that can be legally treated with medical marijuana, the researchers suggest. The study, “Marijuana Use in Adults Living with Sickle Cell Disease,” was published in the journal Cannabis And Cannabinoid Research. In the U.S., the medical use of marijuana to alleviate pain caused by several health conditions is now legal in many states. However, only three states — Connecticut, Ohio, and Pennsylvania — include sickle cell disease as an approved health condition for medical marijuana.  

  Novartis’ investigational therapy for sickle cell disease (SCD), crizanlizumab (SEG101), was seen to reduce the number of patients reporting a pain crisis — including in those with a recent history of multiple crises — a post-hoc analysis of the SUSTAIN Phase 2 study reports. More patients (35.8%) treated with crizanlizumab — among a study subgroup who had between two and 10 vaso-occlusive crises (VOCs) in the year prior to entering the trial — had no such crises post-treatment, compared to those given placebo (16.9%), its researchers reported. These findings were reported in the study, “Effect of crizanlizumab on pain crises in subgroups of patients with sickle cell disease: A SUSTAIN study analysis,” published in the American Journal of Hematology. Crizanlizumab is a monoclonal antibody targeting P-selectin, a molecule existing on the surface of endothelial cells (which line the inner surface of blood vessels), and platelets (cell fragments involved in clotting).