The TMD07 trial demonstrated that the progression to ML-DS cannot be prevented by treating patients with TMD with low-dose cytarabine. However, the prospective setup of the trial revealed the benefit of this well-tolerated treatment for symptomatic children: it significantly reduced deaths in the first six months compared with symptomatic patients in the historical control. This study helped in establishing clear recommendations for the management of children with TMD: patients that present with TMD-related clinical symptoms benefit from low-dose cytarabine treatment to prevent early death. This is now implemented as standard of care in the Princess Máxima Center.