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New drug helps kids with rare immune disease reach Life-Saving transplant

NCT ID NCT02472054

First seen Apr 03, 2026 · Last updated Apr 23, 2026 · Updated 3 times

Summary

This study tested a drug called alemtuzumab as the first treatment for children with a rare, severe immune disorder called hemophagocytic lymphohistiocytosis (HLH). The goal was to see if the drug could keep patients alive long enough to receive a stem cell transplant, which is the only potential long-term control. The study included 29 children and measured how many survived to transplant and how many achieved complete remission.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Locations

  • Hôpital Necker-Enfants Malades

    Paris, 75015, France

Conditions

Explore the condition pages connected to this study.