Once a diagnosis is made, the treatment is based on an individual’s clinical condition and may include standard management for autoimmunity. One patient with severe autoimmune hemolytic anemia responded well to rituximab. Blocking IL-6 activation with tocilizumab in one patient resulted in a dramatic improvement of arthritis. Two patients exhibiting postnatal short<unk> re were treated with growth hormone with good response. Hematopoietic stem cell transplant (HCT) is a possible treatment of this condition, but its effectiveness is unproven. Of the two patients treated by HCT, one patient died and the other was cured of autoimmune symptoms and improved growth. Larger cohorts are required to further validate these therapeutic approaches. Investigators at the National Institute of Allergy and Infectious Diseases at the US National Institutes of Health currently have clinical protocols to study new approaches to the diagnosis and treatment of this disorder.
STAT3 GOF
HL23V
Philadelphia chromosome
Tbx18 transduction
T790M
ABL 
Fusion gene
GcMAF