The University of Minnesota’s Blood and Marrow Transplant (BMT) program performed the world’s first successful BMT in 1968. Since that time, specialists in the program have performed well over 6,000 BMTs. The BMT program at University of Minnesota Masonic Children’s Hospital and the Division of Pediatric Hematology/Oncology work together as a cohesive team, meeting weekly to discuss cases, research, and strategies. Here we describe the complex case of a young girl with a rare type of acute T-lymphoblastic leukemia (ALL) who was successfully co-managed by specialists from both programs.
A 7-year-old girl presented at her local clinic with fever, cough, and bilateral leg pain that was focused in the knees and lower legs. A complete blood count was notable for leukocytosis with an elevated level of blast cells, thrombocytopenia, and anemia (Figures 1 and 2). The patient was admitted to University of Minnesota Masonic Children’s Hospital. She was diagnosed with double pneumonia and a newly described type of ALL, called early T-lymphocyte precursor ALL (ETP-ALL), which carried an extremely poor prognosis.
The patient was treated with standard 4-drug induction chemotherapy; however, evidence of leukemia cells remained after treatment. Because of the poor prognosis associated with ETP-ALL, the patient was considered a candidate for blood and marrow transplant (BMT). Fortunately, her younger brother was an identical HLA match. In preparation for the BMT, a more aggressive chemotherapy regimen was prescribed, and the girl achieved a complete remission. Immediately after, she underwent the BMT procedure.
At 3 years post-transplant, she entered the Cancer Survivor Program at University of Minnesota Masonic Children’s Hospital. The patient is followed at the clinic every 6 months. She has shown no negative cognitive effects from her treatment. Because her treatment resulted in some longterm endocrine deficiencies, she is followed by the endocrinology service at the hospital. The patient has remained in complete remission for >5 years post-BMT. She will receive life-long surveillance for late toxicities of cancer treatment in the Cancer Survivor Program. As she enters adulthood (after age 21), which is also when risks of late-effects start to increase, we will transfer care to our adult specialist partners with University of Minnesota Cancer Care.
ETP-ALL was identified in 2009 as a unique biologic subtype of ALL associated with a very high risk of remission induction failure or relapse in patients treated on standard protocols of intensive chemotherapy.1 Around the time of discovery, when the above case patient was diagnosed, the reported remission failure or hematologic relapse rate was 72% at 10 years. This was dramatically higher than the typical 10% failure rate reported for ALL patients. Gene expression profiling has shown that the leukemic cells in ETP-ALL closely resembled early T-cell precursors, a subgroup of thymocytes which have very recently immigrated from the bone marrow and are closely related to multipotent hematopoietic stem cells. ETP-ALL comprises about 12% of all childhood T-ALL. In line with these data, our approach to these patients often includes BMT in first remission, after consolidation and reintensification therapy, as was indicated for the case patient described above.
1. Coustan-Smith E, Mullighan CG, Onciu M, et al. Early T-cell precursor leukaemia: a subtype of very high-risk acute lymphoblastic leukaemia. Lancet Oncol. 2009;10(2):147-56.
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