Disease-Specific Indications and Outcomes

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Acute Lymphoblastic Leukemia (ALL) - Pediatric

Approximately 6,000 individuals are diagnosed with acute lymphoblastic leukemia (ALL) each year in the United States. Children comprise about 60% of these cases, making ALL the most common type of pediatric cancer. [1]

ALL is the most common indication for allogeneic hematopoietic cell transplantation (HCT) in patients <18 years with hematological malignancies, according to CIBMTR® (Center for International Blood and Marrow Transplant Research®), our research program. [2]

 

Advances

 

Recent research in pediatric ALL, including an evidence-based review [3], provides new data for clinical decision-making and supports the following recommendations:

  • Allogeneic HCT is recommended for pediatric ALL patients who experience primary induction failure, but subsequently achieve a first complete remission (CR1)
  • Allogeneic HCT and intensive chemotherapy with imatinib have equivalent early outcomes for Ph+ ALL in CR1
  • HLA-matched related and unrelated donors provide equivalent outcomes
  • Myeloablative total body irradiation containing conditioning regimens are recommended

Numerous clinical research studies have definitely shown that minimal residual disease (MRD) has a clear prognostic impact in pediatric ALL, and clinicians are increasingly using it for disease risk stratification. [4,5]

Chimeric antigen receptor T cell (CAR-T) therapy for treating relapsed/refractory pediatric and young adult patients with B-cell ALL received FDA approval in 2017. [6]

Outcomes

 

Review outcomes for allogeneic transplantation in children with ALL below. View additional ALL slides showing demographic data and transplant trends.

Data in this section have been prepared by CIBMTR.

Figure 1. ALL Pediatric Survival Over Time, Unrelated HCT in CR1

ALL Pediatric Survival Over Time, Unrelated HCT in CR1
Download slide "ALL Pediatric Survival Over Time, Unrelated HCT in CR1"

 

Figure 2. ALL Pediatric Survival, Unrelated Marrow HCT

ALL Pediatric Survival, Unrelated Marrow HCT
Download slide "ALL Pediatric Survival, Unrelated Marrow HCT"

 

Figure 3. ALL Pediatric Survival, Sibling HCT, by Disease Status

ALL Pediatric Survival, Sibling HCT, by Disease Status
Download slide "ALL Pediatric Survival, Sibling HCT, by Disease Status"

 

Figure 4. ALL Pediatric Survival, Unrelated HCT, by Diseases Status

ALL Pediatric Survival, Unrelated HCT, by Diseases Status
Download slide "ALL Pediatric Survival, Unrelated HCT, by Diseases Status"

Referral Timing Guidelines

 

These guidelines highlight patient and disease characteristics that put patients at risk for disease progression and who should be referred for a consultation for allogeneic transplantation. [7]

High-resolution HLA typing is recommended at diagnosis for all patients

Transplant Consultation Guidelines: Pediatric ALL (age <15 years)

  • Infant at diagnosis
  • Primary induction failure
  • Presence of minimal residual disease after initial therapy
  • High/very high-risk CR1 including:
    • Philadelphia chromosome positive slow-TKI responders or with IKZF1 deletions
    • Philadelphia-like 
    • iAMP21
    • 11q23 rearrangement
  • First relapse
  • CR2 and beyond, if not previously evaluated

Transplant Consultation Guidelines: Pediatric ALL (adolescent and young adults age 15-39 years)

  • Primary induction failure
  • Presence of minimal residual disease after initial therapy
  • High/very high-risk CR1 including:
    • Philadelphia chromosome positive or Philadelphia-like
    • iAMP21
    • 11q23 rearrangement
    • B-cell with poor-risk cytogenetics
  • First relapse
  • CR2 and beyond, if not previously evaluated

Download slides (PPT)
View complete Referral Timing Guidelines

CAR T Cell Therapy Video for Patients

In this easy-to-understand video, Linda J. Burns, M.D., Vice President, Health Services Research, and Scott Kerwin, R.N., M.N., C.C.R.C., C.C.R.N., Clinical Trial Patient Education Specialist, explain what CAR T cell therapy is, who it may help, what the treatment is like, potential risks and benefits, questions to ask your doctor, and more. 

In this easy-to-understand video, Linda J. Burns, M.D., Vice President, Health Services Research, and Scott Kerwin, R.N., M.N., C.C.R.C., C.C.R.N., Clinical Trial Patient Education Specialist, explain what CAR T cell therapy is, who it may help, what the treatment is like, potential risks and benefits, questions to ask your doctor, and more. View the video and share it with patients and caregivers who are looking for information on CAR T therapy as a treatment option.

References

  1. SEER Stat Fact Sheets: Acute Lymphocytic Leukemia. Accessed 1 November, 2017. Access
  2. D'Souza A, Fretham C. Current Uses and Outcomes of Hematopoietic Cell Transplantation (HCT): CIBMTR Summary Slides, 2017. Available at: http://www.cibmtr.org
  3. Oliansky DM, Camitta B, Gaynon P, et al. Role of cytotoxic therapy with hematopoietic stem cell transplantation in the treatment of pediatric acute lymphoblastic leukemia: Update of the 2005 Evidence-Based Review. Biol Blood Marrow Transplant. 2012; 18(4): 505-522. Access
  4. Pui C-H, Pei D, Coustan-Smith E, et al. Clinical utility of sequential minimal residual disease measurements in the context of risk-based therapy in childhood acute lymphoblastic leukaemia: A prospective study. Lancet Oncol. 2015; 16(4): 465-474. Access
  5. Schnittger S. Minimal residual disease monitoring: A new era for childhood ALL. Lancet Oncol. 2015; 16(4): 362-364. Access
  6. Determine Efficacy and Safety of CTL019 in Pediatric Patients With Relapsed and Refractory B-cell ALL (ELIANA) (ClinicalTrials.gov identifier: NCT02435849). Access
  7. NMDP/Be The Match and ASBMT Recommended Timing for Transplant Consultation. Download (PDF)