Patient demographics and clinical characteristics
Fifteen pediatric and young adult patients with hematological
malignancies who received a TPO-RA were included in this case series(Table 1). The cohort consisted of nine females and six males,
with a median age of 16 years (range: 7-25 years). Diagnoses included a
range of hematologic malignancies. Seven patients had undergone a prior
HSCT. Patients received a TPO-RA during a variety of phases of therapy,
including initial induction, consolidation, delayed intensification,
re-induction following relapse, following CAR T-cell therapy and
post-HSCT. Ten patients (67%) had disease in remission at the time of
receiving a TPO-RA.
In 14 patients (93%), a TPO-RA was initiated due to thrombocytopenia
refractory to platelet transfusions. Nine patients (60%) had an
additional comorbidity that placed them at very high risk of bleeding or
refractory thrombocytopenia, including angioinvasive fungal infections
(n=3), history of retinal hemorrhages (n=2), brain abscess (n=1),
transplant-associated thrombotic microangiopathy and respiratory
infections with associated hemoptysis (n=2), and hemorrhagic cystitis
secondary to BK viremia (n=1). One patient was a Jehovah’s Witness who
received romiplostim prophylactically to minimize transfusions. One
patient had refractory thrombocytopenia secondary to human leukocyte
antigen alloimmunization.