Table 2. Details on individual patients... | American Society of Hematology

Table 2.

Details on individual patients and IL-2 course

	Transplant indication	cGVHD features	Previous GVHD therapies∗	Other previous comorbidities	Age at LD IL-2 start (y)	Clinical response (excluding oral/ocular)	Adverse events during therapy (not thought to be from LD IL-2)	Reason for IL-2 dose change or discontinuation
P1	Fanconi anemia	Skin Ocular	Steroid Sirolimus	Acute GVHD: gut	10.6	Overall CR Skin CR		cGVHD stable/improved, thus weaned off therapy
P2	AML	Lung JMF	Steroid Imatinib Ruxolitinib		18.1	Overall PR Lung SD JMF PR	Pulmonary aspergillosis	Reduced to 0.33 dosing because of pain related to injections Stopped because of pulmonary aspergillosis
P3	Mixed leukemia	Liver Ocular Lung†	Steroid Ruxolitinib Vedolizumab	Acute GVHD: skin, oral, gut, liver Stem cell boost for low CD34 counts Eosinophilic gastroenteritis	18.4	Overall PR Liver CR Lung SD	Pulmonary aspergillosis with development of pulmonary GVHD	6.5 mos break after aspergillosis; then restarted LD IL-2; cGVHD stable/improved, thus weaned off therapy
P4	Immune deficiency (WAS)	Lung	Steroid	Initial graft failure followed by second transplant Chronic relapsing encephalomyelitis	4.3	Overall PR Lung PR		cGVHD stable/improved, thus weaned off therapy
P5	fHLH (STXBP2)	Skin Gut Ocular	Steroid Tacrolimus Vedolizumab∗, Ruxolitinib∗	Acute GVHD: skin, gut Recurrent CVL infection Waning chimerism → stem cell boost	4.6	Overall CR Skin CR Gut (not active at time of start)		cGVHD stable/improved, thus weaned off therapy
P6	Omenn syndrome (IL-7R deficiency)	Skin Gut	Steroid	Acute GVHD: gut Feeding intolerance; on parenteral nutrition Central sleep apnea Diabetes CMV and EBV viremia Tracheostomy with recurrent tracheitis and bacteremia	1.2	Overall PR Skin CR Gut PR	Idiopathic giant cell myocarditis	Held during infections & cardiac workup (2 mo) IL-2 eventually paused for trial of mepolizumab (unclear if there would be an interaction)
P7	Immune deficiency (unknown)	Lung	Steroid Cyclosporine MMF	Central nervous system EBV⁺ leiomyosarcoma (resected ×3) Chronic CMV viremia Toxic epidermal necrolysis	8.4	Overall SD Lung SD	Influenza with multifocal bacterial pneumonia	Was initially enrolled on pediatric cohort of LD IL-2 trial and had PR in lungs at week 8. Came off trial at week 97 because of progression in setting of respiratory infection Restarted IL-2 off study 2 mos later. cGVHD stable/improved, thus weaned off therapy
P8	AML	Liver Ocular Oral	Steroid Ibrutinib∗, Infliximab∗	Second transplant after initial relapse of AML Severe malnutrition Organizing pneumonia	18.4	Inevaluable		50% dosing for prerenal AKI Severe liver failure at time of IL-2 start; goals of care soon redirected to comfort measures
P9	ALD	Liver Ocular JMF	Steroid Cyclosporine Rituximab∗, Ibrutinib∗, Ruxolitinib∗		10.2	Overall CR Liver CR JMF CR		Fatigue & flushing; switched to every-other-day dosing; cGVHD stable/improved, thus weaned off therapy
P10	AML	Lung Skin‡	Steroid Cyclosporine Ruxolitinib	Acute GVHD: skin Pulmonary superinfection & scarring HHV-6 & BK Virus	12.7	Overall SD Lung SD	Pseudomonas pneumonia	Insurance stopped covering
P11	SAA	Skin Liver Lung	Steroid Cyclosporine Ruxolitinib	Acute GVHD: liver, gut Thrombocytopenia	10.2	Inevaluable		Stopped IL-2 & ruxolitinib because of thrombocytopenia; later the cause was determined to be immune thrombocytopenia
P12	Gamma-delta T-cell lymphoma	Skin Liver Lung	Steroid	Acute GVHD: liver, gut, skin Polymicrobial infected hemorrhagic pancreatic pseudocyst → distal pancreatectomy, splenectomy, & near-total colectomy Steroid-induced chronic kidney disease	23.2	Overall CR Skin CR Liver CR Lung CR	Pancreatitis	On study for 122 weeks and then continued off-study for 90 weeks cGVHD stable/improved, thus weaned off therapy
P13	Immune deficiency (ITK deficiency)	Skin Gut	Steroid Tacrolimus Vedolizumab	Acute GVHD: skin Liver transplant before HCT for genetic liver failure; complicated by wound dehiscence and intra-abdominal infection requiring abdominal wash out and biliary duct dilation requiring ERCP and stent placement in the common bile duct. Poor graft function despite stem cell boost Persistent HHV-6 viremia Chronic pericardial effusion	4.1	Overall CR Skin CR Gut CR	Bacteremia	Given persistent graft failure, infections, and multiorgan dysfunction, switched to comfort care
P14	B-ALL	Skin Liver Oral	Steroid MMF	Acute GVHD: skin	10.4	Overall PR Skin SD Liver PR		Mild improvement in skin but early plateau; added ruxolitinib & stopped IL-2
P15	Myeloproliferative disorder (JAK2 mutation)	Skin Liver Ocular	Steroid Ruxolitinib	Acute GVHD status unknown Bilateral breast tissue ulceration/sclerodermatous change with MDR PsA that preceded starting IL-2	15.9	Overall PR Skin PR Liver CR	Recurrence of breast tissue infection	Malaise at daily dosing, switched to every other day Discontinued, because unavailable in home country

	Transplant indication	cGVHD features	Previous GVHD therapies∗	Other previous comorbidities	Age at LD IL-2 start (y)	Clinical response (excluding oral/ocular)	Adverse events during therapy (not thought to be from LD IL-2)	Reason for IL-2 dose change or discontinuation
P1	Fanconi anemia	Skin Ocular	Steroid Sirolimus	Acute GVHD: gut	10.6	Overall CR Skin CR		cGVHD stable/improved, thus weaned off therapy
P2	AML	Lung JMF	Steroid Imatinib Ruxolitinib		18.1	Overall PR Lung SD JMF PR	Pulmonary aspergillosis	Reduced to 0.33 dosing because of pain related to injections Stopped because of pulmonary aspergillosis
P3	Mixed leukemia	Liver Ocular Lung†	Steroid Ruxolitinib Vedolizumab	Acute GVHD: skin, oral, gut, liver Stem cell boost for low CD34 counts Eosinophilic gastroenteritis	18.4	Overall PR Liver CR Lung SD	Pulmonary aspergillosis with development of pulmonary GVHD	6.5 mos break after aspergillosis; then restarted LD IL-2; cGVHD stable/improved, thus weaned off therapy
P4	Immune deficiency (WAS)	Lung	Steroid	Initial graft failure followed by second transplant Chronic relapsing encephalomyelitis	4.3	Overall PR Lung PR		cGVHD stable/improved, thus weaned off therapy
P5	fHLH (STXBP2)	Skin Gut Ocular	Steroid Tacrolimus Vedolizumab∗, Ruxolitinib∗	Acute GVHD: skin, gut Recurrent CVL infection Waning chimerism → stem cell boost	4.6	Overall CR Skin CR Gut (not active at time of start)		cGVHD stable/improved, thus weaned off therapy
P6	Omenn syndrome (IL-7R deficiency)	Skin Gut	Steroid	Acute GVHD: gut Feeding intolerance; on parenteral nutrition Central sleep apnea Diabetes CMV and EBV viremia Tracheostomy with recurrent tracheitis and bacteremia	1.2	Overall PR Skin CR Gut PR	Idiopathic giant cell myocarditis	Held during infections & cardiac workup (2 mo) IL-2 eventually paused for trial of mepolizumab (unclear if there would be an interaction)
P7	Immune deficiency (unknown)	Lung	Steroid Cyclosporine MMF	Central nervous system EBV⁺ leiomyosarcoma (resected ×3) Chronic CMV viremia Toxic epidermal necrolysis	8.4	Overall SD Lung SD	Influenza with multifocal bacterial pneumonia	Was initially enrolled on pediatric cohort of LD IL-2 trial and had PR in lungs at week 8. Came off trial at week 97 because of progression in setting of respiratory infection Restarted IL-2 off study 2 mos later. cGVHD stable/improved, thus weaned off therapy
P8	AML	Liver Ocular Oral	Steroid Ibrutinib∗, Infliximab∗	Second transplant after initial relapse of AML Severe malnutrition Organizing pneumonia	18.4	Inevaluable		50% dosing for prerenal AKI Severe liver failure at time of IL-2 start; goals of care soon redirected to comfort measures
P9	ALD	Liver Ocular JMF	Steroid Cyclosporine Rituximab∗, Ibrutinib∗, Ruxolitinib∗		10.2	Overall CR Liver CR JMF CR		Fatigue & flushing; switched to every-other-day dosing; cGVHD stable/improved, thus weaned off therapy
P10	AML	Lung Skin‡	Steroid Cyclosporine Ruxolitinib	Acute GVHD: skin Pulmonary superinfection & scarring HHV-6 & BK Virus	12.7	Overall SD Lung SD	Pseudomonas pneumonia	Insurance stopped covering
P11	SAA	Skin Liver Lung	Steroid Cyclosporine Ruxolitinib	Acute GVHD: liver, gut Thrombocytopenia	10.2	Inevaluable		Stopped IL-2 & ruxolitinib because of thrombocytopenia; later the cause was determined to be immune thrombocytopenia
P12	Gamma-delta T-cell lymphoma	Skin Liver Lung	Steroid	Acute GVHD: liver, gut, skin Polymicrobial infected hemorrhagic pancreatic pseudocyst → distal pancreatectomy, splenectomy, & near-total colectomy Steroid-induced chronic kidney disease	23.2	Overall CR Skin CR Liver CR Lung CR	Pancreatitis	On study for 122 weeks and then continued off-study for 90 weeks cGVHD stable/improved, thus weaned off therapy
P13	Immune deficiency (ITK deficiency)	Skin Gut	Steroid Tacrolimus Vedolizumab	Acute GVHD: skin Liver transplant before HCT for genetic liver failure; complicated by wound dehiscence and intra-abdominal infection requiring abdominal wash out and biliary duct dilation requiring ERCP and stent placement in the common bile duct. Poor graft function despite stem cell boost Persistent HHV-6 viremia Chronic pericardial effusion	4.1	Overall CR Skin CR Gut CR	Bacteremia	Given persistent graft failure, infections, and multiorgan dysfunction, switched to comfort care
P14	B-ALL	Skin Liver Oral	Steroid MMF	Acute GVHD: skin	10.4	Overall PR Skin SD Liver PR		Mild improvement in skin but early plateau; added ruxolitinib & stopped IL-2
P15	Myeloproliferative disorder (JAK2 mutation)	Skin Liver Ocular	Steroid Ruxolitinib	Acute GVHD status unknown Bilateral breast tissue ulceration/sclerodermatous change with MDR PsA that preceded starting IL-2	15.9	Overall PR Skin PR Liver CR	Recurrence of breast tissue infection	Malaise at daily dosing, switched to every other day Discontinued, because unavailable in home country

AKI, acute kidney injury; ALD, adrenoleukodystrophy; AML, acute myeloid leukemia; B-ALL, B-cell acute lymphoblastic leukemia; BK, BK virus; CMV, cytomegalovirus; CVL, central venous line; EBV, Epstein-Barr virus; ERCP, endoscopic retrograde cholangiopancreatography; fHLH, familial hemophagocytic lymphohistiocytosis; HHV-6, human herpesvirus 6; ITK, interleukin-2-inducible T-cell kinase; JMF, joints, muscles, fascia; MDR, multidrug resistant; MMF, mycophenolate mofetil; SAA, severe aplastic anemia; PsA, Pseudomonas; WAS, Wiskott-Aldrich syndrome.

∗

Indicates had been stopped before starting IL-2; otherwise, therapies active at time of IL-2 start.

†

Developed lung involvement after start of IL-2 in the setting of aspergillosis.

‡

Hypopigmentation, vitiligo; not part of official NIH scoring therefore not used for response (although improved).

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