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HSCT access limited by country, cost
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Although the number of hematopoietic stem cell transplants performed worldwide reached 1 million in 2012, the procedure is primarily utilized by patients who reside in wealthier countries with larger donor registries, according to study results.
“The transplantation of cells, tissues and organs has extended the lifespan of hundreds of thousands of patients worldwide and enhanced their quality of life,” Dietger Niederwieser, MD, of the University Hospital Leipzig in Germany, and colleagues wrote. “As part of this success, the availability of products and procedures has decreased; expectations of patients in need have triggered organ trade and stem cell tourism.”
Niederwieser and colleagues evaluated data from the Worldwide Network for Blood and Marrow Transplantation that detailed the use hematopoietic stem cell transplantation (HSCT) procedures and growth of donor registries in 194 WHO member countries since the first transplant was performed in 1957. The researchers also studied the connection between economic factors, such as gross national income and health care expenditure, in relation to transplant frequency per 10 million residents of each country.
Overall, the number of HSCTs performed increased from approximately 10,000 in 1985 to nearly 100,000 in 1995, and then to more than 1 million by 2012.
Between 1987 and 2012, the number of countries with donor registries increased from two to 57, and the number of registered donors increased from 3,072 to more than 22.3 million. The number of cord blood banks also increased from two in 1993 — the year they were first established — to 36 in 2012.
Researchers evaluated data from 953,651 patients who underwent HSCT at one of 1,516 transplant centers from 75 countries. Forty-two percent of these procedures were allogeneic and 58% were autologous.
More than three-quarters of transplants were performed in Europe (53%) and the Americas (31%). Southeast Asia and Western Pacific countries accounted for 15% of transplants and eastern Mediterranean and African countries accounted for 2% of the total transplants.
No transplants occurred in countries with fewer than 300,000 inhabitants, a surface area smaller than 700 km2 or a gross national income less than $1,260 U.S. dollars.
The number of transplants per 10 million people was highest in countries with a greater gross domestic product per person (R2 = .43), greater gross national income per person (R2 = .06), greater health care expenditure per person (R2 = .53), more donors (R2 = .09) and bigger cord blood banks (R2 = .15).
“Patients, many of them children, are facing a life-and-death situation,” Niederwieser and colleagues wrote. “Ultimately, they will die if they cannot get the treatment they need. All countries need to provide adequate infrastructure for patients and donors to make sure that everyone who needs a transplant gets one, rather than the present situation in which access remains restricted to countries and people with sufficient resources.”
In an accompanying editorial, Edward A. Copelan, MD, FACP, chair of the department of hematologic oncology and blood disorders at Levine Cancer Institute at Carolinas HealthCare System and a HemOnc Today editorial board member, suggested means for improving the underuse of HSCT apparent in the study data.
“As results of transplantation continue to improve, and the number of centers and center capacity increases, oversight of global activity is crucial,” Copelan wrote. “HSCT is an expensive procedure requiring up-front investment, complex care and substantial expertise; it is cost-effective only when many procedures are done at an institution. The outcome of HSCT is affected by the center where the transplantation is done; centers in which physicians care for a larger number of transplant patients per year seem to have low mortality compared with those who do fewer transplants.”
Key elements for successful programs include training, well-defined infrastructure, standard operating procedures, strategies for improvement and meaningful quality oversight, Copelan wrote.
“Further, collaborations among centers in data collection and analysis — and especially in performance of prospective studies — are essential to advancing the specialty,” he wrote. “Cooperation of national and regional consortiums with global organizations, exemplified by the Worldwide Network for Blood and Marrow Transplantation, promotes worldwide studies and standards that will help us define the future, including extension of HSCT to more of those in need.” – by Cameron Kelsall
Disclosure: The researchers report no relevant financial disclosures.
Perspective
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Joseph McGuirk, DO
The study by Niederwieser and colleagues is indicative of a remarkable success story in cancer medicine. Stem cell transplantation has now saved hundreds of thousands of lives that would absolutely have been lost. The growth in availability and reliability of this procedure is unprecedented.
Increasingly, we are moving away from very intensive radiation and chemotherapy regimens to make these transplants successful. Because of our better understanding of the immunology of the patients and their donors, the technology of the transplants is expanding in an extraordinary, exponential way. The understanding and manipulation of the molecular machinery of these cells, and the manipulation of the immune system to allow for better treatment reaction, will make these transplants much safer and more effective, and could potentially lead to cost reduction over time.
Moving forward, we need to consider the reality that a substantial part of the world’s population does not have access to this curative treatment modality in any way, shape or form. The United States, Western Europe, Israel and Japan are the places where transplants are readily available to all who might need it. The reasons behind the lack of availability of this treatment in other regions — which include inadequate infrastructure to collect cord blood, diagnostic or laboratory unavailability, and the costly and complex nature of these procedures — should be addressed by WHO.
This study is dominated by data from Europeans, Americans and the Japanese. A large part of the world population is simply left out. Above and beyond many of the scientific questions still in the field, the overwhelming question of availability is significant. With today’s technology, we already have a therapy available that — for hundreds of thousands of people — provides a reasonable chance of cure of the underlying disease and is increasingly well-tolerated, but it is not available for much of the planet.
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