ASH offers early look at updated SCD guidelines: Experts formulated >50 recommendations on sickle cell disease

  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."
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Pfizer rare disease introduces Council for Change to further help SCD patients

  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.  
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Gene therapies could transform the treatment of sickle cell disease

  Manny Johnson keeps catching himself rubbing a spot on the upper right side of his chest. Starting when he was a teenager, the 21-year-old from Boston depended on a port implanted there to ease his monthly blood transfusions for sickle cell disease, a genetic disorder that caused a stroke when he was three. But in November, six months after Johnson became the first patient to receive an experimental therapy aimed at curing his disease, the port that had become part of him — requiring special approval to play sports, used when he was excused from school for a day or two every month for treatments — was removed. Johnson hasn’t needed a transfusion or had any symptoms since May. “It’s like, wow. I’m fascinated by it,” said Johnson, who added that he almost didn’t know what to make of it when his medical team told him they wouldn’t need to see him for three whole months. “I’m so used to being next to them. It’s like: freedom.”  
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Sickle cell patients in UK survey, especially those 16 to 20, voice problems with care and pain relief given

 

In a survey, patients across the U.K. with sickle cell disease — especially adolescents and young adults — voiced discontent with their healthcare experiences, particularly as they relate to emergency care and the promptness of pain relief, a study reports.

The study, “Patient-reported experience measure in sickle cell disease,” was published in the journal Archives of Disease in Childhood.

Sickle cell is among the more common, serious genetic conditions diagnosed in newborns in the U.K., predominantly affecting people of African descent, the researchers note, citing a National Health Service (NHS) report.

Despite its prevalence, understanding and awareness of the condition — which influences patient care — can be poor. Disease symptoms like vaso-occlusive pain crises that strike patients are often treated with opioid-based painkillers that require a prescription, and younger patients requesting such treatment can be regarded with suspicion.

 

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Crizanlizumab lowers pain crises in at-risk sickle cell patients, ad-hoc trial data show

  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).  
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Marijuana use common in sickle cell patients, highlighting need for more research, study shows

  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.  
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School staff lacks knowledge about sickle cell disease, student survey finds

  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.  
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The Sickle Cell Treatment Act (Bill S. 2465) Passed the Senate!

  Senate bill S.2465 (the Sickle Cell Treatment Act) passed the Senate on October 11. The bill is currently in the House and needs your help. Contact your current House Representative before the year is over. Ask them to consider S.2465 for a vote this year. If you don't know how to contact your House member, we've included a handy toolkit with instructions below. This toolkit was created in collaboration with Sick Cells, Sickle Cell 101, The SickleCellForum, #BoldLipsforSickleCell, the EveryLife Foundation, and the Sickle Cell Disease Association of America (SCDAA). The current House still has four weeks (till week of December 10) to work this year. The time between the new House being sworn in and the current House finishing their terms is called lame duck session. Many bills, like S.2465 get passed during this time.
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Doris Duke Charitable Foundation awards grant to Critical Path Institute

 

In a continuing effort to spur advancements in treating and curing sickle cell anemia, the Doris Duke Charitable Foundation today announced a grant to the Critical Path Institute (C-Path) to lay the groundwork for forming a collaboration of multiple stakeholder organizations to accelerate the development of therapies for the disease.

With the funding, C-Path will conduct the planning necessary to bring together scientists, clinicians and representatives from the biopharmaceutical industry, governmental regulatory agencies, academic institutions and patient advocacy organizations to identify and work collaboratively toward solving significant challenges affecting the entire research and development community.

The anticipated outcome is a consortium model engaging potential partners to work on jointly beneficial drug development tools and novel methodologies, submit them to the regulatory agencies for endorsement, and then make them available to the broader research community.

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Presidential Message on National Sickle Cell Disease Awareness Month 2019

National Sickle Cell Disease Awareness Month is a time to acknowledge the challenges faced by Americans with sickle cell disease (SCD) and to reaffirm our commitment to increasing the quality, affordability, and accessibility of different treatment options.

SCD is an inherited red blood cell disorder that is most prevalent among those who have ancestors from sub-Saharan Africa and certain regions of South and Central America. In the United States, SCD occurs in 1 out of every 365 African-American births and 1 out of every 16,300 Hispanic-American births.

Beginning in early childhood, people with SCD experience a lifetime of crippling pain, challenging disabilities, and often premature death. The Centers for Disease Control and Prevention estimates medical expenses of children with SCD average between $11,000 and $14,000, with nearly 40 percent of these patients requiring at least one hospital stay.

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