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What are the indications for a stem cell transplantation (SCT)?

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Key concept

The most common indication for an SCT—specifically, autologous SCT—in the United States remains multiple myeloma. Acute myeloid leukemia (AML), by contrast, is the most common indication for allogeneic SCT.1 The indications for SCTs have continuously been refined by incorporating the cytogenetic composite and biology of tumors, and these are continuously compared against novel therapeutics to determine the safest and most effective approaches.

Clinical scenario

A 47-year-old man is diagnosed with Philadelphia chromosome–negative acute lymphoblastic leukemia (ALL). Cytogenetic analysis reveals a mixed-lineage leukemia translocation. He is treated with modified hyper-CVAD chemotherapy, achieving a complete remission. He relapses 2 months later while on therapy. He receives re-induction therapy with blinatumomab, achieving a second complete remission followed by an allogeneic SCT from a sibling donor who is a 9/10 HLA match.

Action items

The discussion of an SCT should be held with patients and transplant hematologists early during the course of treatment, as soon as it becomes a consideration. The pre-transplant evaluation, and, in allogeneic SCTs, the donor search, can be a lengthy process, and an early referral can prove valuable.


Allogeneic SCT


  • Relapsed/refractory disease

  • Poor-risk disease based on cytogenetics

  • AML with FLT3-ITD mutation2

  • Treatment-related AML (t-AML) unless good risk cytogenetics

  • Secondary AML (eg, secondary to myelodysplastic syndrome or myeloproliferative disorders)


  • Poor-risk ALL based on cytogenetics

  • Minimal residual disease positivity by flow cytometry at the end of induction3

  • Consider if presenting with white blood cell counts >30,000/µL in B-ALL, >100,000/µL in T-ALL, age >30–35, or failure to achieve a complete remission in 4 weeks

Myeloproliferative disorders

  • Blast-phase chronic myeloid leukemia

  • Myelofibrosis: Dynamic international prognostic scoring system-plus intermediate-2 score or high risk, and in high-risk molecular features4

Myelodysplastic syndrome

  • International Prognostic Scoring System intermediate-2 score or high risk5

Bone marrow failure syndromes

  • Aplastic anemia

  • Congenital marrow failure syndromes

Autologous SCT

  • Multiple myeloma

  • Relapsed/refractory diffuse large B cell lymphoma

  • Mantle cell lymphoma in first complete remission6

  • Peripheral T cell lymphoma in first complete remission7

  • Relapsed/refractory Hodgkin lymphoma

  • Relapsed follicular lymphoma

  • Relapsed acute promyelocytic leukemia (APL) if molecular remission is achieved

  • With the exception of APL, acute leukemias require the graft-versus-tumor effect of an allogeneic SCT in cases when an allogeneic SCT is indicated

  • With the exception of ALL, autologous transplantation is used in lymphoid malignancies

  • Owing to the defective stem cells in patients with bone marrow failure syndromes, treatment is with an allogeneic SCT when indicated

  • Autologous SCT can be a non-curative modality to achieve a prolonged progression-free and treatment-free survival

  • The role and sequencing of autologous SCT for multiple myeloma had been and remains a constant topic of discussion among experts, more recently owing to the approval of several anti-myeloma therapeutics

  • Allogeneic SCT can be curative in conditions traditionally treated with autologous SCTs or non-transplant approaches; however, its risks outweigh its benefits

  • Institutions vary ...

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