Girl being weighed in a doctor's office

Stem cell transplant and cycling chemotherapy are two other treatment options for patients with haematologic malignancies, but they have their limitations.1,2

Burden of transplant

Stem cell transplant (SCT) has been a pillar in the treatment of patients with haematologic malignancies, but it is not appropriate for all patients. SCT replaces diseased cells with healthy stem cells from the patient’s own body (autologous) or from a donor (allogeneic).1

Possible side effects and long-term risks of SCT (particularly allogeneic SCT) include the following3:

  • Infections
  • Acute or chronic graft-versus-host disease (GVHD)
  • Organ damage
  • Secondary cancers
  • Infertility

These side effects often require ongoing management and further treatment after transplant.3 To view the side effects of CAR-T cell therapy, please click here.

Graft-versus-host disease

Chronic GVHD remains the most challenging risk factor for patients with haematologic malignancies who have undergone allogeneic SCT.4 Acute or chronic GVHD occurs in 30%-70% of these patients.5 Patients whose donor is unrelated are more likely to develop GVHD than patients whose donor is a compatible sibling.1 GVHD is also associated with additional high-risk conditions.4

Freedom from cycling chemotherapy

Chemotherapy is another treatment option that is helpful for many patients with haematologic malignancies, but relapsed or refractory patients are not likely to achieve lasting remission with this treatment. Chemotherapy is a cycling regimen that interferes with cells’ ability to grow and multiply. This treatment regimen is administered in repeating cycles over a period of weeks, months, or years. The infusion of one dose can take a number of hours.2

The most common side effects of chemotherapy include the following6:

  • Infections
  • Hypersensitivity7
  • Central and/or peripheral neuropathy
  • Bone toxicities
  • Thromboembolism
  • Endocrinopathies
  • Nephrotoxicity

To view the side effects of CAR-T cell therapy, please click here.


Infections are the most frequent complications of chemotherapy and the greatest cause of treatment-related mortality.8 Tolerance to chemotherapy in patients varies widely.9

The value of CAR-T cell therapy

Given that CAR-T cell therapy is a one-time treatment after which patients may not need another therapy, it aims to deliver long-term value to patients and society.10-12

Icon of check mark badge

Better value: CAR-T cell therapy has been shown to be more effective than other treatments for haematologic malignancies, providing improvements in health outcomes for approved indications.13

Icon of first aid kit

Comparable to transplant: CAR-T cell therapy offers a costeffectiveness comparable to standard of care treatment options like SCT.12 Aggregate allogeneic SCT costs are particularly high, as they include inpatient hospitalization for treatment administration, donor costs, management of possible long-term side effects, and potential treatmentrelated mortality.14

Icon of paper money

Societal savings: From a societal perspective, CAR-T cell therapy is expected to result in long-term cost saving by reducing overall healthcare utilisation and non-drug costs.11,15,16 Paediatric cancers have additional costs associated with caregivers that contribute to the aggregate high-cost burden of treating this patient population.12 Successful treatment with CAR-T cell therapy may allow patients and their caregivers to enter or return to the workforce and contribute to society.


To learn more broadly about the difference between cell and gene therapy and standard of care therapy, please click here.


  1. Locatelli F, Zecca M, Messina C, et al. Improvement over time in outcome for children with acute lymphoblastic leukemia in second remission given hematopoietic stem cell transplantation from unrelated donors. Leukemia. 2002;16:2228-2237.
  2. American Cancer Society. Typical treatment of acute lymphoblastic leukemia (ALL). treating/typical-treatment.html(link is external). Accessed May 6, 2019.
  3. American Cancer Society. Stem cell transplant side effects. is external). Accessed April 22, 2019.
  4. Mohty B, Mohty M. Long-term complications and side effects after allogeneic hematopoietic stem cell transplantation: an update. Blood Cancer J. 2011;1(1). doi:10.1038/bcj.2011.14.
  5. Atilla E, Atilla PA, Toprak SK, Demirer T. A review of late complications of allogeneic hematopoietic stem cell transplantations. Clin Transplant. 2017;31(10). is external).
  6. Schmiegelow K, Müller K, Mogensen SS, et al. Non-infectious chemotherapy-associated acute toxicities during childhood acute lymphoblastic leukemia therapy. F1000Res. 2017;6:444. doi:10.12688/f1000research.10768.1.
  7. Hijiya N, van der Sluis IM. Asparaginase-associated toxicity in children with acute lymphoblastic leukemia. Leuk Lymphoma. 2016;57(4):748-757.
  8. Hough R, Vora A. Crisis management in the treatment of childhood acute lymphoblastic leukemia: putting right what can go wrong (emergency complications of disease and treatment). Am Soc Hematol Educ Program. 2017(1);251-258.
  9. Schmiegelow K. Treatment-related toxicities in children with acute lymphoblastic leukaemia predisposition syndromes. Euro J of Med Genetics. 2016;59:654-660.
  10. Kymriah Summary of Product Characteristics. Novartis Pharma AG; 2018.
  11. Cavallo J. Weighing the cost and value of CAR T-cell therapy, a roundtable discussion. Huntington, NY: The ASCO Post; May 25, 2018.
  12. Leech AA, Dusetzina SB. Cost-effective but unaffordable: the CAR-T conundrum [published online ahead of print December 17, 2018]. J Natl Cancer Inst. doi:10.1093/jnci/djy195.
  13. Baumgardner J, Everson K, Brauer M, et al. CAR-T therapy displays favorable gains in health outcomes and competitive cost-effectiveness when compared with past innovative cancer treatments. Poster presented at: 60th American Society of Hematology Annual Meeting & Exposition; December 2, 2018; San Diego, CA.
  14. Broder MS, Quock TP, Chang, E, et al. The cost of hematopoietic stem-cell transplantation in the United States. Am Health Drug Benefits. 2017;10(7):366-379.
  15. Hollmann S, Painter C, Hogan A, et al. Budget impact analysis of tisagenlecleucel for the treatment of paediatric and young adult patients with relapsed or refractory B-cell acute lymphoblastic leukaemia in England. Poster presented at: ISPOR Europe Conference; November 10-14, 2018; Barcelona, Spain.
  16. Hollmann S, Painter C, Hogan A, et al. Budget impact analysis of tisagenlecleucel for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma in England. Poster presented at: ISPOR Europe Conference; November 10-14, 2018; Barcelona, Spain.