Published in October 2022, the French National Diagnostic and Care Protocol (PNDS) for haematopoietic stem cell transplantation in autoimmune diseases includes myasthenia among the indications for this exceptional treatment, under four conditions:
- a diagnosis of proven myasthenia according to the diagnostic criteria of the PNDS Myasthenia,
- an age ≤ 65 years,
- severe myasthenia, class III according to the clinical score of the Myasthenia Gravis Foundation of America (MGFA),
- failure of first and second line treatment with well-conducted immunosuppressants.
However, no mention is made of the type of autoantibodies. A team from Calgary (Canada) reports the case of three patients with myasthenia gravis with anti-MuSK, refractory to several treatments including rituximab:
- between 2017 and 2020, they received an autologous haematopoietic stem cell transplant on average 3.3 years after the diagnosis of myasthenia,
- this treatment led to a major and lasting improvement in the impact of the disease on activities of daily living (average MG-ADL score reduced from 14.7 to 0.3) and on their quality of life (average MG-QoL score reduced from 15 to 0), even though none of them is still taking medication for myasthenia,
- the post-transplant events were mainly treatable infections and early menopause.
Greffe de cellules souches hématopoïétiques dans les maladies auto-immunes – Protocole national de diagnostic et de soins. (in French) Mathec – FAI2R – MaRIH. Octobre 2022
