On Location ASH’s Sickle Cell Disease Summit
• In older children
and young adults,
that includes
vaso-occlusive pain
episodes, acute
chest syndrome,
priapism, retinopathy, and gallstones
• n adults, that inI
cludes skin ulcers,
pulmonary hypertension, sickle
lung disease, renal
insufficiency, and
cardiac dysfunction
Dr. Thompson urged
development of SCD
toolkits and Webbased applications for
emergency personnel,
as well as for advanced
Dr. Charles Abrams leads a breakout session on SCD research initiatives in the United States.
practice practitioners
to raise their readiness
to intervene – especially for SCD pain management. She
The complexities of SCD management, Dr. Thompson
also suggested education to help patients interface more
underscored, vary across a patient’s lifespan.
effectively with the health-care providers they meet when
they experience SCD events.
• n toddlers and young children, that includes handI
Patients are aware that primary-care physicians have
foot syndrome, infection and sequestration pertainlimited SCD knowledge and limited experience with
ing to infancy, vaso-occlusive pain episodes, acute
managing SCD, noted John Strouse, MD, PhD, from
chest syndrome, and brain injuries for toddlers
Johns Hopkins Children’s Center in Baltimore, Maryland,
through roughly the onset of puberty
and a member of the Summit Steering Committee. They
also perceive that communication between primary-care
physicians and hematologists is poor. Urgent appointments are difficult to schedule and follow-up after hospitalizations is poor.2
SIDEBAR
ASH Priorities for Sickle Cell
Disease and Sickle Cell Trait
Research Priorities
•
Identify predictors of disease severity
•
Optimize the use of existing therapies
•
Develop novel therapies
•
Strengthen curative therapies
•
Enhance pain research
•
Improve access to evidence-based care through
innovative health-care delivery models
•
Determine the effects of quality of care on
quality of life
•
Investment in sickle cell trait research
Other Priorities
•
Expand global initiatives
•
Support a sustainable SCD workforce
To learn more about each of these priorities, go to
www.hematology.org/Research/Recommendations/
Sickle-Cell. For additional information and to share
your thoughts and comments, please contact
ASH Government Relations and Practice Manager,
Stephanie Kaplan, at [email protected] or
202-776-0544.
54
ASH Clinical News
Where Do We Need More Information?
Dr. Williams underscored the need for new curative therapies in his presentation, “Research Priorities in SCD.”
“Future care will depend on advanced and highly targeted
approaches to research, discovery, and implementation,” he said. This topic did not end when the Summit
came to a close. It continues to be on ASH’s radar. ASH
will consider updates to “ASH Priorities for Sickle Cell
Disease and Sickle Cell Trait” (SIDEBAR). Over the next few
months, the Society will also further develop the ideas
discussed at the Summit and identify other stakeholders
who need to be involved to help advance these efforts.
Genetic approaches, including gene therapies in
which a functioning gene is inserted into the genome and
gene editing, which repairs the mutated sickle cell beta
gene, head the list of research into curative approaches. A
gene therapy clinical trial is currently opening and a gene
editing trial in thalassemia is slated for this year. Both are
first-in-human trials.
Research is targeted toward hematopoietic cell transplant protocols with less toxicity and less tendency to
cause graft-versus-host disease.
Alleviating symptom burden is also a major area of
investigation; this includes strategies to inhibit the attachment of cells to blood vessel walls and other anti-inflammatory approaches to increase the protective expression
of hemoglobin F.
“These are novel non-curative treatments that should
be supported,” Dr. Williams said. Further research into
SCD pain, an additional high priority, will encompass
basic investigation into neurotransmitters, acute and
chronic SCD pain, psychosocial and environmental
contributors to pain, and biomarkers for response to pain
and opioids.
Discussing the need for reliable biomarkers, Dr. Williams noted that currently there are no methods for determining which children born with SCD will be among
the 50 to 60 percent with no major problems or the 10
percent who are devastated by the disease.
“Developing predictors would be enormously helpful
because we could focus therapeutic resources on those
who most need it, while avoiding exposing patients to
difficult and toxic therapies who do well without them,”
he added.
Quality indicators, Dr. Strouse said, have been
established for individuals younger than 18 years by the
National Quality Measures Clearinghouse3 but not for
adults. Proposed measurement items discussed at the
Summit include: the proportion of patients with hemoglobin sickle syndrome treated with hydroxyurea; time
to first parenteral analgesic for severe pain; the proportion of patients readmitted within 30 days of hospital
discharge; and immunizations, retinopathy, renal disease,
and iron overload screening.
Funding and Reimbursement
“If we had adequate funding from government, private
foundations, and industry, we could make huge inroads
in SCD treatment,” Dr. Abrams said. The underuse of hydroxyurea, for one, could be addressed through education
and relevant research, which requires increased funding.
Payers, naturally, may well be interested in and influenced by such research. Low reimbursement for treating
SCD patients can also affect quality of care, according to
Dr. Thompson.
“Primary-care providers are challenged every day with
large volumes of patients whose insurance sources provide
inadequate reimbursement, given the time that needs to
be invested to effectively manage this complex, chronic
illness,” she said. “We recognize that some fundamental
changes in SCD reimbursement may be needed.”
Referencing the large number of SCD patients who
have non-private insurance, Dr. Thompson noted that a
representative from the Centers for Medicare & Medicaid
Services participated in the Summit. “To find achievable
solutions, we need to bring them and other insurers to
the table.”
How to allocate funds across the range of clinical and
logistical aspects of SCD care is a complex problem, Dr.
Thompson acknowledged. “I applaud ASH’s willingness
and ability to take a leadership role. ASH is well-suited for
this role and can definitely make some headway.”
When asked about his impressions of the Summit, Dr.
Williams responded, “I was delighted to hear, multiple
times, that participants really valued and appreciated
ASH’s initiative in this arena.”
Summing up, Dr. Williams said that the major challenges are improving access to care, advancing research,
and addressing the global disparities in outcomes for
individuals with SCD. “This is a large undertaking and
truly a call to action for ASH. It means taking leadership,
identifying other partners at the federal level and from the
private sector, and bringing them tog ether to look at tough
questions – and to come up with durable answers.” ●
REFERENCES
1. Brousseau DC, Owens PL, Mosso AL, et al. Acute care utilization and rehospitalizations for
sickle cell disease. JAMA 2010;303:1288-1294.
2. Liem RI, O’Suoji C, Kingsberry PS, et al. Access to patient-centered medical homes in children
with sickle cell disease. Matern Child Health J. 2014;18:1854-62.
3. Agency for Healthcare Research and Quality. Sickle Cell Disease Measures from the National
Quality Measures Clearinghouse. Accessed April 30, 2015 from http://www.qualitymeasures.
ahrq.gov/browse/by-organization-indiv.aspx?objid=47883.
May 2015