Inotuzumab ozogamicin in infants and young children with relapsed or refractory acute lymphoblastic leukaemia: a case

Erica Brivio1,2, Christophe F Chantrain3, Tanja A Gruber4,5

  • 1Princess Máxima Center for Pediatric Oncology, Utrecht, the Netherlands.

Insights

Inotuzumab ozogamicin shows promise for infant acute lymphoblastic leukemia (ALL). This study found a 47% remission rate in young children with relapsed or refractory ALL, suggesting further research is warranted.

Area of Science:

  • Pediatric Oncology
  • Hematology
  • Immunotherapy

Background:

  • Infant acute lymphoblastic leukemia (ALL) is rare and aggressive.
  • Limited treatment options exist for relapsed or refractory (R/R) infant ALL.
  • No prior data on inotuzumab ozogamicin (InO) in this population.

Purpose of the Study:

  • To evaluate the efficacy and safety of InO in infants and young children (<3 years) with R/R ALL.
  • To assess remission rates and overall survival.
  • To identify potential toxicities associated with InO treatment.

Main Methods:

  • International data collection on 15 infant/young children with ALL treated with InO.
  • Patients had R/R disease, median age 4.4 months.
  • Median CD22+ blast percentage was 72%; median InO dose 1.74 mg/m².

Main Results:

  • Seven patients (47%) achieved complete remission (CR).
  • One additional patient converted from minimal residual disease (MRD)-positive to MRD-negative.
  • Six-month overall survival was 47%; two patients experienced veno-occlusive disease post-transplant.

Conclusions:

  • Inotuzumab ozogamicin demonstrates potential efficacy in infant R/R ALL.
  • Further investigation of InO in this specific pediatric ALL subgroup is justified.
  • Careful monitoring for adverse events like veno-occlusive disease is necessary.

Related Concept Videos

Treatment Resistant Cancers02:56

Treatment Resistant Cancers

Cancer is the second leading cause of death in the United States. A cancer cell is genetically unstable and hence can mutate faster. They can also modify their microenvironment and escape immune surveillance. The difficulties in treating cancer are further compounded by the emergence of rapid resistance to anticancer drugs. The most common ways to attain resistance in cancer cells include alteration in drug transport and metabolism, modification of drug target, elevated DNA damage response, or...
3.5K
Drug Dosing: Infants and Children01:29

Drug Dosing: Infants and Children

Pediatric patient dosages diverge from adults due to disparities in body surface area, total body water, and extracellular fluid per kilogram of body weight. The dosing regimen considers the variations in pharmacokinetics and pharmacology across distinct age groups, encompassing preterm newborns, infants, young children, older children, and adolescents. Calculation of pediatric patient doses is predicated on determining body surface area, which exhibits a superior correlation with the child's...
102
Pharmacokinetics in Pediatric Patients: Overview and Drug Absorption01:23

Pharmacokinetics in Pediatric Patients: Overview and Drug Absorption

Understanding the physiological differences in the pediatric population is crucial for effective pharmacotherapy. Neonates, infants, and children exhibit significant variations in gastric pH, gastric emptying time, intestinal transit time, and biliary function. These variations profoundly affect oral drug absorption, necessitating a nuanced approach to pediatric dosing.Neonates present with a unique physiological profile, having a gastric pH greater than 4 and faster and more irregular gastric...
102
Pharmacokinetics in Pediatric Patients: Drug Metabolism01:24

Pharmacokinetics in Pediatric Patients: Drug Metabolism

In pediatric care, understanding the nuances of hepatic drug metabolism is crucial, as it significantly differs from that of adults. This divergence is primarily due to the developmental stage of drug-metabolizing enzymes, which affects how medications are processed in the body. In neonates, for instance, the activity of Phase I enzymes—critical for the initial breakdown of drugs—is markedly reduced, functioning at just 20–40% of the levels seen in adults. This reduction poses...
89
Pharmacokinetics in Pediatric Patients: Drug Distribution01:17

Pharmacokinetics in Pediatric Patients: Drug Distribution

Drug distribution in the pediatric population exhibits unique challenges and considerations due to the physiological differences between children, particularly neonates and infants, and adults. A crucial aspect of pediatric pharmacology is understanding how these differences impact the pharmacokinetics of various drugs, necessitating age-specific dosing strategies to ensure efficacy and safety.Neonates and infants have a higher total body water content, ~75%–90% of their body weight,...
127