Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome

• Defibrotide (Drug), also known as Defitelio®: Defibrotide 6.25 mg/kg IV q6h up to 7 days

Primary outcomes

• Number of patients with grade III/IV allergic reaction to defibrotide [30 days]: All patients will be monitored for allergic reaction probably or definitely related to defibrotide administration.
• Number of patients with grade III/IV hemorrhage [30 days]: All patients will be monitored for hemorrhage probably or definitely related to defibrotide.

Secondary outcomes

• Number of patients with improvement in clinical signs of Acute Chest Syndrome [30 days]: Patients will have pre and post treatment assessments with imaging and blood tests to monitor clinical signs of ACS

Sex: All, ages: 2 Years to 40 Years

Inclusion Criteria:

* SCD-associated ACS with the presence of any two or more of the following signs not explained by other etiologies: Fever, Chest pain, Cough, Dyspnea, Tachypnea for age, Pulmonary infiltrate on CXR and/or Chest CT scan, Decreased O2 saturation with or without oxygen supplement;
* Age 2 to 40 years of age;
* Homozygous Hemoglobin S Disease, Hemoglobin SC Disease or Hemoglobin S 0/+ thalassemia;
* Informed consent/assent;
* Consent of patient/parent within ≤72 hours after inpatient admission for SCD-associated ACS.
* Females of childbearing age will have a negative pregnancy test.

Exclusion Criteria:

* Current Grade III or IV hemorrhage;
* Previous hypersensitivity reaction to defibrotide;
* Current systemic anti-coagulant therapy and/or fibrinolytic therapy;
* Consent of patient/parent greater than 72 hours of inpatient admission for SCD-associated ACS;
* No signed informed consent

• Mitchell S Cairo, MD, Principal investigator, New York Medical College