علاج فقر الدم فانكوني مع فشل نخاع العظم حديث الظهور في المراهقين والبالغين الشباب

References

DOI: 10.1182/hematology.2023000489

Any AYA patient (up to age 40 years) who presents with BMF or MM associated with 1q, 3q, or 7q copy number abnormalities/translocations or unusual solid tumors for age (oral, head/neck, genital) should undergo a diagnostic evaluation for FA.

In contrast, use of these supportive strategies is reasonable for new-onset BMF occurring in AYA patients, as these individuals are already at high age-based risk for HSCT complications.

While early data on use of metformin and eltrombopag hold promise, data supporting the use of androgen therapy are the most extensive.

Indeed, AYA patients with FA may exhibit stable hematopoietic function on androgen therapy for many years.