ASH Clinical News ACN_4.14_Full Issue_web | Page 26

Pulling Back the Curtain: Alexis Thompson, MD, MPH I was working with other investigators who were making seminal observations in the laboratory about the pathophysiology of sickle cell disease and looking at new molecular tools to be able to characterize new forms of beta thalassemia, which is another inherited blood disorder caused by mutations in the same gene as sickle cell disease. After fellowship I further honed my skills in the laboratory, working with Randolph Wall, PhD, at the University of California in Los Angeles (UCLA) and learned so much from his graduate students and postdocs. I appreciated the support I received from Stephen Feig, MD, my division chief, that allowed me to get a stronger foothold in the lab. The culture of scientific discovery at the UCLA Jonsson Comprehensive Cancer Center and in the clinical programs were so nurturing. “I’ve worked with many students in my laboratory or acted as a mentor ... , largely because I know I benefited from those relationships and guidance.” Throughout your career, I’m sure you’ve had opportunities to mentor younger hematologists in training. What advice do you share with them as they start their careers? I strongly recommend that they focus first on their craft, whether in the lab or at the bedside, and taking themselves, their careers, and their skills seriously. When I entered medicine, there were so many things that I wanted to do to change the world immediately; I advise younger trainees to stay focused. Later in their careers, they can expand, but at least early on, becoming excellent in one area will allow them to be 24 ASH Clinical News more effective in the long run. I also strongly encourage them to find mentors as they begin to take on more and more interests and projects. It’s the challenge of harnessing their excitement and energy at the beginning of their careers – it certainly inspires me! One of the opportunities that I was able to take advantage of while I was a junior faculty member was receiving funding through the Robert Wood Johnson Foundation’s Minority Medical Faculty Development Program, which gave me a four-year period of financial support and protected research time. Eventually, that program spawned the ASH-Harold Amos Medical Faculty Development Program (AMFDP), which was renamed and expanded in honor of Harold Amos, PhD, who was the first African-American to chair a department at Harvard Medical School. Knowing that I benefited personally early in my career from having a lab mentor and other career mentors through that program, I became a strong advocate in support of initiatives like the ASH- AMFDP and Minority Medical Student Awards Program. I’ve worked with many students in my laboratory or acted as a mentor for their clinical research or for career development, largely because I know I benefited from having those BESPONSA™ (inotuzumab ozogamicin) is indicated for the treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL) AIM FOR DEEP REMISSION Deep remission refers to MRD-negative remission, defi ned in the INO-VATE ALL study as leukemic cells comprising <1 x 10 -4 of bone marrow nucleated cells per fl ow cytometry. 1 The effi cacy of BESPONSA was established on the basis of CR (35.8% [39/109; 95% CI, 26.8-45.5] with BESPONSA vs 17.4% [19/109; 95% CI, 10.8-25.9] with SC), the duration of CR (8.0 months [95% CI, 4.9-10.4] with BESPONSA vs 4.9 months [95% CI, 2.9-7.2] with SC), and the proportion of MRD-negative CR (89.7% [35/39; 95% CI, 75.8-97.1] with BESPONSA vs 31.6% [6/19; 95% CI, 12.6-56.6] with SC) in the fi rst 218 randomized patients. 1 IMPORTANT SAFETY INFORMATION WARNING: HEPATOTOXICITY, INCLUDING HEPATIC VENO-OCCLUSIVE DISEASE (VOD) (ALSO KNOWN AS SINUSOIDAL OBSTRUCTION SYNDROME) and INCREASED RISK OF POST–HEMATOPOIETIC STEM CELL TRANSPLANT (HSCT) NON-RELAPSE MORTALITY (NRM): • Hepatotoxicity, including fatal and life-threatening VOD, occurred in patients who received BESPONSA. The risk of VOD was greater in patients who underwent HSCT after BESPONSA treatment. The use of HSCT conditioning regimens containing 2 alkylating agents and last total bilirubin ≥ upper limit of normal (ULN) before HSCT were signifi cantly associated with an increased risk of VOD • Other risk factors for VOD in patients treated with BESPONSA included ongoing or prior liver disease, prior HSCT, increased age, later salvage lines, and a greater number of BESPONSA treatment cycles • Elevation of liver tests may require dosing interruption, dose reduction, or permanent discontinuation of BESPONSA. Permanently discontinue treatment if VOD occurs. If severe VOD occurs, treat according to standard medical practice • There was a higher post-HSCT non-relapse mortality rate in patients receiving BESPONSA, resulting in a higher Day 100 post-HSCT mortality rate Hepatotoxicity, Including Hepatic VOD: Hepatotoxicity, including fatal and life-threatening VOD, occurred in 23/164 patients (14%) during or following treatment with BESPONSA or following subsequent HSCT. VOD was reported up to 56 days after the last dose during treatment or follow-up without an intervening HSCT. The median time from HSCT to onset of VOD was 15 days. Patients with prior VOD or serious ongoing liver disease are at an increased risk of worsening liver disease, including development of VOD, following treatment with BESPONSA. Monitor closely for signs and symptoms of VOD; these may include elevations in total bilirubin, hepatomegaly (which may be painful), rapid weight gain, and ascites. For patients proceeding to HSCT, the recommended duration of treatment with BESPONSA is 2 cycles. A third cycle may be considered for patients who do not achieve a CR or CRi and MRD-negativity after 2 cycles. Monitor liver tests closely during the fi rst month post HSCT, then less frequently thereafter, according to standard medical practice. Grade 3/4 increases in aspartate aminotransferase, alanine aminotransferase, and total bilirubin occurred in 7/160 (4%), 7/161 (4%), and 8/161 (5%) patients, respectively. Increased Risk of Post-HSCT Non-Relapse Mortality (NRM): There was a higher post-HSCT NRM rate in patients receiving BESPONSA, resulting in a higher Day 100 post-HSCT mortality rate. The rate of post-HSCT NRM was 31/79 (39%) with BESPONSA and 8/35 (23%) with investigator’s choice of chemotherapy. In the BESPONSA arm, the most common causes of post- HSCT NRM included VOD and infections. Monitor closely for toxicities post HSCT, including signs and symptoms of infection and VOD. Myelosuppression: Myelosuppression, and severe, life-threatening, and fatal complications of myelosuppression, including hemorrhagic events and infections, have occurred with BESPONSA. Thrombocytopenia and neutropenia were reported in 83/164 patients (51%) and 81/164 patients (49%), respectively. Febrile neutropenia was reported in 43/164 patients (26%). Monitor complete blood counts prior to each dose of BESPONSA and monitor for signs and symptoms of infection, bleeding/hemorrhage, or other effects of myelosuppression during treatment and provide appropriate management. As appropriate, administer prophylactic anti-infectives during and after treatment with BESPONSA. Dose interruption, dose reduction, or permanent discontinuation may be required.