Persisting clones in compartments other than bone marrow may not be covered by MRD quantification but could still be responsive to blinatumomab therapy. The responses detected in this TCF3-HLF-positive ALL cohort are encouraging and suggest that the application of immunotherapy prior to extensive clonal selection secondary to intensive chemotherapy may be beneficial. As data are updated, the true value of this approach can be assessed. The benefit of adding blinatumomab to frontline ALL chemotherapy will be addressed in an international prospective clinical trial (clinicaltrials.gov identifier: 03643276). Taken together, our results indicate that immunotherapy may improve the outcome of TCF3-HLF-positive ALL.

The agreement between the HJHS scores and the MRI scores suggests that the HJHS may be used safely as a first-line tool. We recommend that the FISH should be used in the routine follow-up of hemophilia patients as a functional evaluation tool. Painful joints may be useful in deciding to apply MRI, whereas the bleeding frequency may not be useful.

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The regression and relapse rates were 100% and 33% for SS-LCH. The regression and relapse rates were 73%, and 18% for MS-LCH. Two infants with MS-LCH died despite chemotherapy.Pulmonary and liver involvements affected outcome adversely in MS-LCH. Multidisciplinary treatment approaches are needed.

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