InO - A Retrospective Study of UK Patients With Leukaemia

Completed

Conditions: Precursor Cell Lymphoblastic Leukemia-Lymphoma

Interventions: Drug: Inotuzumab Ozogamicin

Registration Number: NCT04456959

Lead Sponsor: Pfizer

Brief Summary: The purpose of this study is to describe the demographics and clinical characteristics, treatment pathway, and effectiveness and safety of inotuzumab ozogamicin in patients with relapsed/refractory B-cell acute lymphoblastic leukaemia treated with inotuzumab ozogamicin in the real-world.

Detailed Description: Not available

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 28

Inclusion Criteria

Patients with relapsed/refractory ALL.
Patients who initiated InO between 1st of June 2016 and date of data collection.
Patients who accessed InO treatment via NHS commissioning, via the CUP, or via private purchase.
Patient aged ≥18 years old at initiation of InO treatment

Exclusion Criteria

Patients initiated on treatment with InO at a different hospital than the ones selected in this study.
Patients with <3 months of follow-up since index date, unless death occurs <3 months from index date.

Study & Design

Study Type: OBSERVATIONAL

Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Adult R/R ALL patients who have received InO	Inotuzumab Ozogamicin	Relapsed/refractory ALL patients who are 18 years and over and initiated InO between 1st of June 2016 and date of data collection (to be confirmed). They will have accessed InO treatment via NHS commissioning, via the CUP, or via private purchase and will have at least 3 months follow up from the index date unless death occurs within that time.

Primary Outcome Measures

Name	Time	Method
Number of Participants According to Number of Lines of Salvage Therapy	Anytime between initial diagnosis of ALL and InO initiation, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	In this outcome measure, number of participants according to number of lines of salvage therapy anytime between initial diagnosis of ALL and InO initiation, were reported.
Number of Participants According to Type of Conditioning Regimen for Each HSCT	Anytime between initial diagnosis of ALL and InO initiation, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	In this outcome measure, number of participants were classified according to different type of conditioning regimen for each HSCT (high-dose intensity myeloablative, reduced-intensity/non-myeloablative), were reported.
Number of Participants Who Received First Line Chemotherapy According to National Trial or Treatment Guideline	Anytime between initial diagnosis of ALL and InO initiation, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	In this outcome measure, number of participants who were treated with the first-line chemotherapy during anytime between initial diagnosis of ALL and InO initiation, were reported.
Number of Participants Treated With Chimeric Antigen Receptor (CAR) T-Cell Therapies	Anytime between initial diagnosis of ALL and InO initiation, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	In this outcome measure, number of participants treated with chimeric antigen receptor (CAR) T-cell therapies before initiation of InO, were reported.
Number of Participants According to Prior Hematopoietic Stem Cell Transplant (HSCT)	Anytime between initial diagnosis of ALL and InO initiation, during data identification period from June 2016 to January 2021 (approximately 4.5 years); retrieved data was analyzed during 12 months of this observational study	In this outcome measure, number of participants, who were treated with hematopoietic stem cell transplant (HSCT) before initiation of InO, were reported.
Number of Participants Who Were Treated Previously With Blinatumomab	Anytime between initial diagnosis of ALL and InO initiation, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	In this outcome measure, number of participants who were previously treated with blinatumomab, were reported.

Secondary Outcome Measures

Name	Time	Method
Number of Participants Classified on the Basis of Any Modifications in Inotuzumab Ozogamicin Dose	From InO initiation date to date of end of treatment, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	In this outcome measure, number of participants were classified as following: 1) with no dose modification and 2) no data recorded.
Overall Survival (OS)	InO initiation date to death due to any cause or last visit at time of data collection, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	OS was defined as the time from the index date to the date of death. Participants were censored at date of latest visit at the time of data collection. Kaplan-Meier method was used for OS analysis.
Number of Participants According to Types of Therapies Post Inotuzumab Ozogamicin Treatment	Post InO treatment, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	In this outcome measure, number of participants according to therapies they initiated post InO treatment were reported. One participant could have more than 1 type of therapies.
Number of Participants Who Experienced a Documented Diagnosis of Veno-occlusive Disease (VOD)/Sinusoidal Obstruction Syndrome (SOS) Post InO Treatment	Post InO treatment, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	VOD, also called SOS, happens when the small blood vessels that lead into the liver and are inside the liver become blocked.
Number of Participants Who Were Treated With Concomitant Azole Antifungal Therapy	From InO initiation date to date of end of treatment, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	In this outcome measure, number of participants who were treated with concomitant azole antifungal therapy along with InO treatment were reported.
Duration of Concomitant Azole Antifungal Therapy	From InO initiation date to date of end of treatment, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	In this outcome measure, time/duration between start date and end date of concomitant azole antifungal, was reported.
Number of Participants Who Achieved Complete Remission (CR) by the End of InO Treatment	From InO initiation date to date of end of treatment, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	CR was defined as documented in medical records or (if unavailable in the records) as less than (\<) 5% blasts in the bone marrow and the absence of peripheral blood leukemic blasts, full recovery of peripheral blood counts (platelets greater than or equal to \[\>=\] 100\10\^9 cells per liter \[/L\] and absolute neutrophil counts \[ANC\] \>=1\10\^9 cells/L) and resolution of any extramedullary disease.
Total Duration of Treatment With Inotuzumab Ozogamicin	From InO initiation date to date of end of treatment, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	In this outcome measure, total duration of InO treatment was reported.
Number of Participants Who Achieved CR With Incomplete Hematological Recovery (CRi) by the End of InO Treatment	From InO initiation date to date of end of treatment, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	CRi was defined as documented in medical records or (if unavailable in the records) \<5% blasts in the bone marrow and the absence of peripheral blood leukemic blasts, incomplete recovery of peripheral blood counts (platelets \<100\* 10\^9 cells/L and ANC \<1\*10\^9 cells/L) and resolution of any extramedullary disease.
Median Time to CR/CRi	From InO initiation date to date of end of treatment, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	CR was defined as documented in medical records or as \<5% blasts in the bone marrow and the absence of peripheral blood leukemic blasts, full recovery of peripheral blood counts (platelets \>=100\10\^9 cells/L and ANC \>=1\10\^9 cells/L) and resolution of any extramedullary disease. CRi was defined as documented in medical records or \<5% blasts in the bone marrow and the absence of peripheral blood leukemic blasts, incomplete recovery of peripheral blood counts (platelets \<100\10\^9 cells/L and ANC \<1\10\^9 cells/L) and resolution of any extramedullary disease.
Number of Participants Who Achieved Negative Minimal Residual Disease (MRD) Among Those Who Had CR/CRi	From InO initiation date to date of end of treatment, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	Negative MRD was defined as documented in medical records or (if unavailable in the records) as leukemic cells comprising \<1\10\^-4 (\<0.01%) of bone marrow nucleated cells. This outcome measure was analyzed in participants with CR/CRi. CR was defined as documented in medical records or as \<5% blasts in the bone marrow and the absence of peripheral blood leukemic blasts, full recovery of peripheral blood counts (platelets \>=100\10\^9 cells/L and ANC \>=1\10\^9 cells/L) and resolution of any extramedullary disease. CRi was defined as documented in medical records or \<5% blasts in the bone marrow and the absence of peripheral blood leukemic blasts, incomplete recovery of peripheral blood counts (platelets \<100\10\^9 cells/L and ANC \<1\*10\^9 cells/L) and resolution of any extramedullary disease.
Number of Participants With CR/CRi by the End of InO Treatment	From InO initiation date to date of end of treatment, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	In this outcome, number of participants who achieved CR/CRi at the end of InO treatment are reported. CR was defined as documented in medical records or as \<5% blasts in the bone marrow and the absence of peripheral blood leukemic blasts, full recovery of peripheral blood counts (platelets \>=100\10\^9 cells/L and ANC \>=1\10\^9 cells/L) and resolution of any extramedullary disease. CRi was defined as documented in medical records or \<5% blasts in the bone marrow and the absence of peripheral blood leukemic blasts, incomplete recovery of peripheral blood counts (platelets \<100\10\^9 cells/L and ANC \<1\10\^9 cells/L) and resolution of any extramedullary disease.
Number of Participants Who Experienced Grade 3 and Grade 4 (Lung/Cardiac/Kidney/Liver) Treatment Related Adverse Event (TRAE) Following Inotuzumab Ozogamicin Initiation	From InO initiation date to date of end of treatment, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	Adverse event (AE) was defined as any untoward medical occurrence in participant who received study drug without regard to possibility of causal relationship. Grade 3 were severe events. Grade 4 were life-threatening events. Information for grades was recorded as per participants' medical records.
Number of Participants According to Reasons for Inotuzumab Ozogamicin Treatment Interruption	From InO initiation date to date of end of treatment, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	In this outcome measure, number of participants, were reported according to reasons of interruption in respective Cycles.
Number of Participants According to Prescribed Inotuzumab Ozogamicin Doses	From InO initiation date to date of end of treatment, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	In this outcome measure, number of participants according to prescribed starting InO dose, were reported.
Number of Participants Who Survived Following Treatment For Documented Diagnoses of Veno-occlusive Disease (VOD)/Sinusoidal Obstruction Syndrome (SOS)	Post InO treatment, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	VOD, also called SOS, happens when the small blood vessels that lead into the liver and are inside the liver become blocked.
Number of Participants Who Achieved Negative MRD Classified Per InO Cycles	From InO initiation date to date of end of treatment, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	Negative MRD (among those who had CR/CRi) was defined as documented in medical records or (if unavailable in the records) as leukemic cells comprising \<1\10\^-4 (\<0.01%) of bone marrow nucleated cells. CR was defined as documented in medical records or as \<5% blasts in the bone marrow and the absence of peripheral blood leukemic blasts, full recovery of peripheral blood counts (platelets \>=100\10\^9 cells/L and ANC \>=1\10\^9 cells/L) and resolution of any extramedullary disease. CRi was defined as documented in medical records or \<5% blasts in the bone marrow and the absence of peripheral blood leukemic blasts, incomplete recovery of peripheral blood counts (platelets \<100\10\^9 cells/L and ANC \<1\*10\^9 cells/L) and resolution of any extramedullary disease.
Number of Participants Who Survived at 3, 6 and 12 Months Post InO Treatment Initiation	At 3, 6, and 12 months post InO initiation date, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	In this outcome measure, number of participants who survived 3, 6, and 12 post InO treatment, were reported.
Number of Participants Who Survived Post InO Blinatumomab Treatment	Post InO treatment, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	In this outcome measure, number of participants who survived at completion of InO treatment were reported.
Number of Participants According to Type of Treatments Received for Documented Diagnoses of Veno-occlusive Disease (VOD)/Sinusoidal Obstruction Syndrome (SOS)	Post InO treatment, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	VOD, also called SOS, happens when the small blood vessels that lead into the liver and are inside the liver become blocked.
Number of Participants With Interrupted InO Treatment Due to VOD/SOS	Post InO treatment, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	VOD, also called SOS, happens when the small blood vessels that lead into the liver and are inside the liver become blocked.
Number of Participants With Moderate Severity VOD/SOS	Post InO Treatment, during data identification period from June 2016 to January 2021 (approximately 4.5 years); from the data collected and observed retrospectively over approximately 12 months of this study	VOD, also called SOS, happens when the small blood vessels that lead into the liver and are inside the liver become blocked.
Number of Participants According to Types of Treatments Received for Grade3/4 TRAE Following Inotuzumab Ozogamicin Initiation	From InO initiation date to date of end of treatment, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	AE was defined as any untoward medical occurrence in participant who received study drug without regard to possibility of causal relationship. Grade 3 were severe events. Grade 4 were life-threatening events. Information for grades was recorded as per participants' medical records.
Percentage of Participants Who Were Relapse-free at 3, 6 and 12 Months Post InO Treatment Initiation	At 3, 6, and 12 months from InO initiation date, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	Relapse free survival: the time from the start of treatment to earliest date of the following events: death, progressive disease (including objective progression, relapse from CR/CRi, treatment discontinuation due to global deterioration of health status), and start of new induction therapy or post-therapy HSCT without achieving CR/CRi; as documented in medical records. CR: documented in medical records or as \<5% blasts in the bone marrow and the absence of peripheral blood leukemic blasts, full recovery of peripheral blood counts (platelets \>=100\10\^9/L and ANC \>=1\10\^9/L) and resolution of any extramedullary disease. CRi: documented in medical records or \<5% blasts in the bone marrow and the absence of peripheral blood leukemic blasts, incomplete recovery of peripheral blood counts (platelets \<100\10\^9/L and ANC \<1\10\^9/L) and resolution of any extramedullary disease. Progressive disease (PD): a doubling of peripheral blasts with an absolute increase of \>5\*10\^9 cells/L.
Number of Participants According to Number of Inotuzumab Ozogamicin Treatment Cycles	From InO initiation date to date of end of treatment, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	In this outcome measure, number of participants were classified according to total number of InO treatment cycles received.
Number of Participants According to Interrupted Inotuzumab Ozogamicin Treatment Cycles	From InO initiation date to date of end of treatment, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	In this outcome measure, number of participants were classified according to number of interrupted cycles of InO treatment.
Number of Participants Classified According to Their Cause of Death	From InO initiation date to date of end of treatment, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	In this outcome measure, number of participants according to their cause of death were reported.
Relapse-free Survival (RFS)	From InO initiation date to death or progressive disease, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	RFS was defined as the time from the start of treatment to earliest date of the following events: death, PD (including objective progression, relapse from CR/CRi, treatment discontinuation due to global deterioration of health status), and start of new induction therapy or post-therapy HSCT without achieving CR/CRi; as documented in medical records. CR was defined as documented in medical records or as \<5% blasts in the bone marrow and the absence of peripheral blood leukemic blasts, full recovery of peripheral blood counts (platelets \>=100\10\^9 cells /L and ANC \>=1\10\^9 cells/L) and resolution of any extramedullary disease. CRi was defined as documented in medical records or \<5% blasts in the bone marrow and the absence of peripheral blood leukemic blasts, incomplete recovery of peripheral blood counts (platelets \<100\10\^9 cells/L and ANC \<1\10\^9 cells/L) and resolution of any extramedullary disease. PD: a doubling of peripheral blasts with an absolute increase of \>5\*10\^9 cells/L.
Time to Non-relapse Mortality (NRM)	Post InO treatment from date of follow up HSCT to death, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	NRM was defined as the time from the date of follow-up HSCT until death due to any cause without disease progression or relapse.
Number of Participants Who Achieved CR, CRi, Progressive Disease and Stable Disease With Different Types of Post Inotuzumab Ozogamicin Treatments	Post InO treatment, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	CR was defined as documented in medical records or as \<5% blasts in the bone marrow and the absence of peripheral blood leukemic blasts, full recovery of peripheral blood counts (platelets \>=100\10\^9 cells/L and ANC \>=1\10\^9 cells/L) and resolution of any extramedullary disease. CRi was defined as documented in medical records or \<5% blasts in the bone marrow and the absence of peripheral blood leukemic blasts, incomplete recovery of peripheral blood counts (platelets \<100\10\^9 cells/L and ANC \<1\10\^9 cells/L) and resolution of any extramedullary disease. PD was defined as a doubling of peripheral blasts with an absolute increase of \>5\*10\^9 cells/L. Stable disease was defined as increase of peripheral blasts with an absolute increase not \>50%.
Number of Participants With Peripheral Blood Blast Counts Measurement Prior to Post InO HSCT	Prior to post InO HSCT, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study
Number of Participants With Liver Dysfunction Following Inotuzumab Ozogamicin Initiation	From InO initiation date to date of end of treatment, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study
Number of Participants With Significant Risk Factors for VOD/SOS	From InO initiation date to date of end of treatment, during data identification period from June 2016 to January 2021 (approximately 4.7 years); retrieved data was analyzed during 12 months of this observational study	In this outcome measure, participants with significant risk factor for VOD/ SOS occurrence were reported. VOD, also called SOS, happens when the small blood vessels that lead into the liver and are inside the liver become blocked

Trial Locations

Locations (4): University College London Hospital NHS Foundation Trust
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London, United Kingdom
University Hospitals Bristol NHS Foundation Trust
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Bristol, United Kingdom
The Royal Marsden NHS Foundation Trust of Fulham Road
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London, United Kingdom
Taunton and Somerset NHS Foundation Trust of Musgrove Park Hospital
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Taunton, United Kingdom