Science Stories

“We understand the deep benefit that prolonged survival offers patients and their loved ones, which is why we remain committed to promoting research, education and clinical practice in the transplantation field.” – Dr. Navneet Majhail, MD, MS, President, ASTCT

For many people with blood cancer, HSCT can provide a meaningful option for prolonged survival.

How Stem Cell Transplantation Can Improve Outcomes for Patients With Blood Cancers

Every three minutes, someone in the U.S. is diagnosed with a blood cancer.1 Fast-growing blood cancers like acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML) can become life-threatening if left untreated because they can quickly spread to other parts of the body, including the brain and spinal cord.2,3 Hematopoietic stem cell transplantation (HSCT) can provide an important option in improving outcomes for appropriate patients.4,5,6

In most cases of ALL and AML, chemotherapy is the primary treatment because it is the most aggressive way to stop or slow the growth of cancer.7,8,9,10 Yet while chemotherapy can be effective in killing cancer cells, it also destroys healthy cells in the process.10 For certain patients, HSCT is an attractive option because it replaces the blood-forming cells that were damaged by chemotherapy.11

HSCT involves extracting stem cells from the blood or bone marrow of a donor, which can be a relative or non-relative; older patients are more likely to find a match on the National Bone Marrow Registry, rather than in a relative.11 Once acquired from a donor, the healthy cells are infused into the patient’s body through a central venous catheter, similar to a blood transfusion.3 These stem cells then grow into and restore the body’s blood cells.3

While age and comorbidities factor into a patient’s eligibility for HSCT, the increasing availability of stem cell donors and improved identification of appropriate patients have helped increase the safety and feasibility of this treatment option.12

The American Society for Transplantation and Cellular Therapy (ASTCT) is an international professional membership association focused on promoting research, education and clinical practice in the transplantation and cellular therapy field. The organization advocates for the practice of HSCT in certain patients with malignant hematologic disorders like ALL and AML, and has identified stem cell biology and transplantation as top research priorities.13

“While there are many factors – such as a patient’s age, overall health and donor type – to consider before transplant, it can be a particularly attractive option for certain patients with blood cancer,” said Dr. Navneet Majhail, MD, MS, President, ASTCT. “We understand the deep benefit that prolonged survival offers patients and their loved ones, which is why we remain committed to promoting research, education and clinical practice in the transplantation field.”

At Jazz, we know that one of the only things worse than learning that you or a loved has a disease is learning that there are limited or no options to treat it. While we’ve made significant strides in advancing the science behind managing these diseases, we recognize there is still work to be done in addressing patients’ needs. We’re committed to changing that through ongoing research and disease education programs.


1. Leukemia Research Foundation. Statistics on Leukemia and Other Blood Cancers. https://allbloodcancers.org/statistics/. Accessed January 14, 2020.
2. American Cancer Society. What is Acute Lymphocytic Leukemia (ALL)? https://www.cancer.org/cancer/acute-lymphocytic-leukemia/about/what-is-all.html. January 16, 2020.
3. National Cancer Institute. Adult Acute Myeloid Leukemia Treatment (PDQ) – Patient Version. https://www.cancer.gov/types/leukemia/patient/adult-aml-treatment-pdq. Accessed January 16, 2020.
4. American Cancer Society. High-dose Chemotherapy and Stem Cell Transplant for Childhood Leukemia. https://www.cancer.org/cancer/leukemia-in-children/treating/bone-marrow.html. Accessed January 14, 2020.
5. American Cancer Society. Stem Cell Transplant for Acute Lymphocytic Leukemia (ALL). https://www.cancer.org/cancer/acute-lymphocytic-leukemia/treating/bone-marrow-stem-cell.html. Accessed January 14, 2020.
6. American Cancer Society. Stem Cell Transplant for Acute Myeloid Leukemia (AML). https://www.cancer.org/cancer/acute-myeloid-leukemia/treating/bone-marrow-stem-cell-transplant.html. January 14, 2020.
7. American Cancer Society. Treatment of Children with Acute Lymphocytic Leukemia (ALL). https://www.cancer.org/cancer/leukemia-in-children/treating/children-with-all.html. Accessed January 14, 2020.
8. American Cancer Society. Typical Treatment of Acute Lymphocytic Leukemia (ALL). https://www.cancer.org/cancer/acute-lymphocytic-leukemia/treating/typical-treatment.html. Accessed January 14, 2020.
9. American Cancer Society. Typical Treatment of Acute Myeloid Leukemia (Except APL). https://www.cancer.org/cancer/acute-myeloid-leukemia/treating/typical-treatment-of-aml.html. Accessed January 14, 2020.
10. National Cancer Institute. Chemotherapy to Treat Cancer. https://www.cancer.gov/about-cancer/treatment/types/chemotherapy. Accessed January 14, 2020.
11. MDS Foundation. Expert Video – What is a stem cell transplant? Who is a candidate for it? http://www.youandaml.com/en-aml/view/m401-e403-what-is-a-stem-cell-transplant-who-is-a-candidate-for-it-expert-video Accessed January 14, 2020.
12. Döhner H, Estey E, Grimwade D, et al. Diagnosis and management of AML in adults: 2017 ELN recommendations from an international expert panel. Blood. 2017;129(4):424-447.
13. Research Priorities. ASTCT. https://www.astct.org/about/research-priorities. Accessed December 2019.