Stem Cell Transplants: Underuse Still a Problem

Veronica Hackethal, MD

March 05, 2015

The use of hematopoietic stem cell transplantation (HSCT) throughout the world has expanded since the first bone marrow transplant took place more than 50 years ago. By the end of 2012, HSCTs had reached the 1 million mark, and they had been performed in 75 countries. However, wide variations and the underuse of HSCTs exist in some areas, likely because of resource shortages, according to a study published online February 27 in the Lancet Haemotology.

"The frequency of stem cell transplantation is highest in developed countries, although there is considerable heterogeneity worldwide," said lead author Dietger Niederwieser, MD, professor of medicine at the University of Leipzig in Germany. "It is essential that practicing physicians have dedicated transplant centers cooperating with them. Early referral and adequate follow-up of patients is essential. Too many patients die because of late referral and because of insufficient transplant beds."

"Today, stem cell transplantation is often the only curative treatment for hematologic diseases and several types of cancer," Dr Niederwieser explained. "Stem cell transplantation is also the most cost-effective treatment — especially for congenital hematologic disorders such as sickle cell anemia and thalassemia — compared with lifelong transfusion treatment."

HSCT began as experimental bone marrow procedure more than 50 years ago. Two types of HSCT exist: autologous (which transplants the patient's own healthy cells) and allogeneic (which uses cells from a donor or, since 1988, from umbilical cord blood).

For their review, Dr Niederwieser and colleagues used data from 194 World Health Organization (WHO) member states collected by the Worldwide Network for Blood and Marrow Transplantation (WBMT) from January 2006 to December 2014. They looked at factors such as healthcare expenditures and gross national income per capita to see how these affect the frequency of HSCT.

Results showed a large expansion in the use of HSCT. From 1957 (when the first transplant took place) to 1985, the use rose to 10,000. By 1995, this had climbed to 100,000. Ten years later, in 2005, the use had risen to 500,000. By 2012, the use had doubled, to 1 million (42% allogeneic and 58% autologous). By the end of 2012, HSCTs were being performed in 75 countries and 1516 transplant centers around the world.

The most HSCTs took place in Europe (501,315), the Americas (296,754), Southeast Asia and the Western Pacific (140, 079), and the Eastern Mediterranean and Africa (15,503).

The use of allogeneic HSCTs increased around the world, but with wide variation in different regions, ranging from 0.4 to 506 per 10 million inhabitants. Allogeneic HSCTs were most common in countries with higher gross national incomes per capita and in countries with the most HSCTs overall — namely, France, Germany, Italy, Japan, and the United States.

There was a trend toward saturation of autologous HSCTs in Europe and the Americas, suggesting the underuse of allogeneic HSCTs and perhaps the overuse of autologous HSCTs, the authors note.

From 1987 to 2012, the number of countries with registries increased from two to 57, and the number of registered donors increased from 3072 to 22,346,551.

Over time, the use of unrelated donors increased, surpassing family donors in 2006. The international exchange of stem cell products also increased; there were 10,000 exchanges annually from 2006 to 2012. However, the quantity of imported and exported stem cells varied widely between countries.

The results suggest that international differences in the frequency and spread of HSCT are "mainly" the result of macroeconomic factors related to economic development and infrastructure. The proportion of HSCTs was higher in countries with a higher gross domestic product per capita, gross national income per capita, healthcare expenditure per person, and Human Development Index. It was also higher in countries with more donors and bigger cord blood banks.

Although the cost of HSCT varies considerably in different countries, the authors did not find any resistance to such an expensive treatment, even in poor countries. However, they note that quality-control measures and adequate infrastructure, such as local transplant centers and donor registries, need to be established.

"Collaboration with WHO and politicians will help to alleviate this problem. The WBMT, in cooperation with the WHO, has started workshops in the low-activity countries to sensitize health politicians and to help them twin with expert centers for setting up or optimizing current programs," Dr Niederwieser reported. "We also need the help of practicing physicians to recruit voluntary donors around the world."

"These data document the extreme range in the present use of transplants between countries and regions with greater resources and those that are poor," writes Edward Copelan, MD, FACP, chair of the Department of Hematologic Oncology and Blood Disorders at the Levine Cancer Institute in Charlotte, North Carolina, in an accompanying comment.

Transplantation is still underused, even in countries with many transplant centers, such as the United States, Dr Copelan noted.

"Disparities in referral patterns to transplant centers based on age, insurance coverage, and race have been demonstrated and are areas requiring intervention," he explained.

Dr Copelan emphasized the "vital role" of collaboratives like the WBMT, because data collection and studies can help define global unmet needs and identify solutions.

"The number of hematopoietic stem cell transplants performed annually is growing rapidly and results continue to improve significantly," Dr Copelan concludes. This research "elegantly demonstrates how detailed study of past practice has led to significant advances in present practice and can identify areas requiring further improvement."

Dr Niederwieser and Dr Copelan have disclosed no relevant financial relationships.

Lancet Haematol. Published online February 27, 2015. Abstract, Comment

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