علاج فقر الدم فانكوني مع فشل نقي العظم الحاد عندما ينطوي الزرع المكوّن للدم على خطر وفاة استثنائي (العمر ≥35، أو خلل وظائف الأعضاء، أو ورم خبيث)

References

If blood counts decline to severe levels (Table 1) and cure by HCT is not possible or preferred, alternative therapies may maintain blood counts and quality of life in patients with FA.

Patients with an exceptional risk of HCT-related mortality (e.g., patients with severe organ dysfunction, those who are 35 years or older, and those with pre-existing malignancies or life-threatening systemic infections) may consider alternative treatment options first, such as the use of androgens.

Synthetic androgens, such as oxymetholone and danazol, have been used to treat cytopenias in patients with FA for more than 50 years.

Most patients who will respond do so within three to four months with stabilization of falling counts or an increase in the hemoglobin or platelet counts.