An infant was diagnosed with severe hemophilia A after developing splenic injury (injury to the spleen), a rare condition among newborns. For the first time, however, a newborn with these conditions was successfully treated with recombinant factor VIII replacement therapy without the need for surgical intervention, doctors reported.
The report, titled “Successful Medical Management Of A Neonate With Spontaneous Splenic Rupture And Severe Hemophilia A,” was published in the journal Hematology Oncology and Stem Cell Therapy.
Spontaneous rupture of the spleen during the newborn period is very rare and usually associated with an underlying coagulation disorder or abnormalities of the spleen. In most cases, splenic rupture occurs due to increased intrathoracic pressure during delivery, which puts the newborn’s spleen at risk of injury during labor.
In normal conditions, the body triggers a response to stop spleen bleeding, but in patients with blood coagulation disorders such as hemophilia, the spleen may rupture hours to days later.
The newborn boy was born after an unremarkable delivery. His family had no history of bleeding problems. But four days after his birth, he was admitted to the ER with worsening anemia and was later diagnosed with spontaneous splenic rupture. His symptoms included abdominal distension with a slightly enlarged liver, tachycardia (accelerated heart beat), poor feeding, and worsening yellow skin color.
The patient then received a blood transfusion and was treated with recombinant factor VIII concentrate (rFVIII, a therapy for hemophilia A) at a dose of 50 IU/kg every eight hours. He continually received infusions to maintain FVIII activity near 100 percent for 72 hours.
At 12 days of age, the infant received another blood transfusion because his response to the initial transfusion was not satisfactory, indicating ongoing bleeding, but he remained stable throughout the process.
After the second transfusion, the infant presented less abdominal distension and discomfort, and at 13 days of age he was restarted on enteral feeding (feeding directly through the stomach), which was well tolerated. The frequency of rFVIII infusions was decreased to every 12 hours for the next 48 hours, and then to every 24 hours for the next three days. He was discharged home when he was 18 days old.
“The hemophilia team followed the patient in the outpatient bleeding disorders clinic with further reduction in his rFVIII infusions to every 48 [hours] for 7 days, and then transitioned him to an on-demand infusion regimen,” researchers wrote. “He continued to be followed in our comprehensive hemophilia clinic every 6 months and currently receives on-demand rFVIII replacement therapy.”
According to the doctors, the infant has received a total of 17 doses of rFVIII (3,692 IU) with no evidence of inhibitors, and continues to grow and develop normally.
“Based on our literature review, we have reported on the case of the first neonate with spontaneous splenic rupture and severe hemophilia A who underwent successful medical treatment without surgical intervention,” the doctors wrote. “In newborns with hemophilia or other bleeding disorders, appropriate replacement of exogenous factor concentrates could prevent emergency splenectomy.”