There is good evidence favoring allogeneic SCT in, Philadelphia chromosome–negative myeloproliferative neoplasm (Ph- MPN.’ however, the event-free survival rate of patients with pediatric MDS and MPN is still only approximately 60%). There are two types of factors that are the major drivers determining patient outcome with SCT: 1) patient factors such as age and the presence or absence of other medical problems (“comorbid conditions”), and 2) disease factors. Patients who are younger, lack other medical problems besides the MPN, and are able to be active (“good performance score”) do best with stem cell transplant. Those who have high WBC, high circulating blasts, high risk chromosome profile, and other negative disease factors tend to do more poorly.
A large retrospective study by Lussana et al confirms that transplantation is potentially curative for patients with end-stage polycythemia vera/essential thrombocythemia progressing to myelofibrosis or acute myeloid leukemia.
Müller LP, Müller-Tidow C. The indications for allogeneic stem cell transplantation in myeloid malignancies. Dtsch Arztebl Int. 2015;112(15):262–270. doi:10.3238/arztebl.2015.0262