Autologous stem cell transplantation in chronic myeloid leukaemia: A meta-analysis of six randomized trials

Rationale A number of collaborative trial groups developed prospective randomized trials to compare autologous stem cell transplantation (ASCT) with non-transplant therapy (interferon-α alone or in combination) for chronic myeloid leukaemia (CML) with the aim of obtaining reliable evidence on the possible benefit of ASCT. With the arrival of tyrosine kinase inhibitors, notably imatinib, these trials closed early without reaching their recruitment targets and no trial was able to address its objectives. Following discussions with the principal investigators, it was agreed that a meta-analysis be performed to attempt to determine the effect of ASCT on the main outcomes. Objectives To establish the effect of ASCT followed by interferon-αcompared with interferon-α only. Findings There was no evidence of a difference in survival; odds ratio = 0.99 (95% confidence intervals = 0.67–1.46). Nor were there statistically significant differences between treatment groups in best haematological or cytogenetic response achieved in the first year. It was not possible to analyse whether autografting with predominantly Philadelphia negative cells early on in the disease resulted in a better outcome. Conclusions The results do not suggest a role for ASCT in initial treatment for CML, but it may still merit investigation in patients resistant to tyrosine kinase inhibitors.