Prospective Observational Study Of Patients With Polycythemia Vera In US Clinical Practices (REVEAL)

Sponsor

Incyte Corporation (Industry)

Overall Status

Completed

CT.gov ID

NCT02252159

Collaborator

(none)

2,544

Enrollment

266

Locations

60.1

Actual Duration (Months)

9.6

Patients Per Site

0.2

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This is a Phase IV, multicenter, non-interventional, non-randomized, prospective, observational study in an adult population (patients >18 years old) of men and women who have been diagnosed with clinically overt PV and are being followed in either community or academic medical centers in the United States who will be enrolled over a 12-month period and observed for 36 months from the date the last patient is enrolled.

Condition or Disease	Intervention/Treatment	Phase
MPN (Myeloproliferative Neoplasms)

Study Design

Study Type:

Observational

Actual Enrollment :

2544 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

Prospective, Non-interventional Study of Disease Progression and Treatment of Patients With Polycythemia Vera in United States Academic or Community Clinical Practices

Actual Study Start Date :

Jul 31, 2014

Actual Primary Completion Date :

Aug 3, 2019

Actual Study Completion Date :

Aug 3, 2019

Arms and Interventions

Arm	Intervention/Treatment
Cohort A Patients with clinically overt PV (and not exhibiting any of the characteristics listed for Cohort B), managed with: Watchful waiting (with or without aspirin), or Phlebotomy (PHL) alone (with or without aspirin) - or HU alone (without concomitant PHL, with or without aspirin). (*Unless patient has a history of intolerance or clinical resistance/ refractoriness to hydroxyurea [HU] (as assessed by the treating physician) - in which case, s/he belongs to Cohort B)
Cohort B Patients with clinically overt PV, with one or more of the following disease characteristics: Treatment with HU and PHL in combination or Treatment with any agent other than HU or aspirin (e.g., recombinant interferon (IFN) or pegylated IFN preparations, busulfan, anagrelide) or A history of thrombosis (venous or arterial) or A history of intolerance or clinical resistance/ refractoriness to HU (as assessed by the treating physician) or Presence of documented splenomegaly (clinically assessed by palpation) or Presence of one or more of the following uncontrolled symptoms related to PV despite therapy (Symptoms deemed uncontrolled as per physician's judgment) Tiredness Difficulty sleeping Itching Muscle aches and/or bone pain Night sweats Sweats while awake Other

Arm

Intervention/Treatment

Cohort A

Patients with clinically overt PV (and not exhibiting any of the characteristics listed for Cohort B), managed with: Watchful waiting (with or without aspirin)*, or Phlebotomy (PHL) alone (with or without aspirin)* - or HU alone (without concomitant PHL, with or without aspirin). (*Unless patient has a history of intolerance or clinical resistance/ refractoriness to hydroxyurea [HU] (as assessed by the treating physician) - in which case, s/he belongs to Cohort B)

Cohort B

Patients with clinically overt PV, with one or more of the following disease characteristics: Treatment with HU and PHL in combination or Treatment with any agent other than HU or aspirin (e.g., recombinant interferon (IFN) or pegylated IFN preparations, busulfan, anagrelide) or A history of thrombosis (venous or arterial) or A history of intolerance or clinical resistance/ refractoriness to HU (as assessed by the treating physician) or Presence of documented splenomegaly (clinically assessed by palpation) or Presence of one or more of the following uncontrolled symptoms related to PV despite therapy (Symptoms deemed uncontrolled as per physician's judgment) Tiredness Difficulty sleeping Itching Muscle aches and/or bone pain Night sweats Sweats while awake Other

Outcome Measures

Primary Outcome Measures

Description of patterns of the disease Polycythemia Vera (PV), and associations of such patterns with patients' exposures or treatments [Every 3 months for approximately 36 months]
Incidence (frequency) of disease progression [Every 3 months for approximately 36 months]
Incidence (frequency) of healthcare resources utilization [Every 3 months for approximately 36 months]
Healthcare resources were defined as a. Medical visits (including Office visits, ER visits, and hospitalizations), b. phlebotomy (PHL) procedures and c. prescriptions (including PV-related prescriptions, PV-related over-the-counter (OTC) medications, and prescription medications for co-morbid conditions).
Incidence (frequency) of complications due to PV [Every 3 months for approximately 36 months]
Incidence (frequency) and description of PV-related symptoms [Every 3 months for approximately 36 months]

Secondary Outcome Measures

Incidence (frequency) of adverse events (for those patients on active therapies, including PHL) [Baseline through end of study. Approximately 36 months]
Patient-reported outcomes as assessed by Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) for assessment of the severity of symptoms [Every 3 months for approximately 36 months]
The MPN-SAF TSS is a validated 10-item instrument that efficiently assesses the prevalence and severity of PV symptoms in both clinical practice and trial settings. Higher scores indicate more severe symptoms and greater inactivity with a possible total score range of 0-100.
Work Productivity and Activity Impairment Questionnaire - Specific Health Problem (WPAI-SHP) score for assessment of work productivity and activity impairment [Every 3 months for approximately 36 months]
WPAI-SHP is a validated 6-item instrument that measures the effect of overall health and specific symptoms on productivity at work and outside of it. All outcomes are presented as a proportion, with 0% representing minimal/no impairment and 100% representing maximal impairment or productivity loss.
European Organization for Research and Treatment of Cancer Core Quality of Life Questionnaire (EORTC QLQ-C30) for Health Related Quality of Life (HRQoL) [Every 3 months for approximately 36 months]
Caregiver burden [Every 3 months for approximately 36 months]
The amount of health care received by a paid healthcare worker or by an unpaid caregiver
Burden of phlebotomy (PHL) [Every 3 months for approximately 36 months]
A patients self-reported assessment of burden including pain at site, difficulty and inconvenience of scheduling and cost factors.

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Age ≥18 years
Diagnosis of Polycythemia Vera (PV)
Willing and able to provide written informed consent
Willing and able to complete patient assessment questionnaires either alone or with minimal assistance from a caregiver and/or trained site personnel
Under the supervision of a physician for the current care of PV including but not limited to watchful waiting, acetylsalicylic acid (ASA) 81mg or greater, antithrombotic therapy, Phlebotomy (PHL), Hydroxyurea (HU), interferon (recombinant or pegylated), busulfan, anagrelide

Exclusion Criteria:

Participation in an active clinical trial in which the study treatment is blinded
Life expectancy <6 months
Diagnosis of myelofibrosis (MF) [including primary MF, post-PV MF, or post-essential thrombocythemia MF (post-ET MF)]
Diagnosis of secondary Acute Myeloid Leukemia (AML)
Diagnosis of Myelodysplastic Syndrome (MDS)
History of or active plan to proceed to allogeneic hematopoietic stem cell transplant in next 3 months
Splenectomy

Contacts and Locations

Locations

	City	State	Country
1	Green Valley	Arizona	United States
2	Safford	Arizona	United States
3	Scottsdale	Arizona	United States
4	Tucson	Arizona	United States
5	Yuma	Arizona	United States
6	Jonesboro	Arkansas	United States
7	Little Rock	Arkansas	United States
8	North Little Rock	Arkansas	United States
9	Stuttgart	Arkansas	United States
10	Berkeley	California	United States
11	Burbank	California	United States
12	Corona	California	United States
13	Fountain Valley	California	United States
14	Highland	California	United States
15	Laguna Hills	California	United States
16	Los Alamitos	California	United States
17	Oceanside	California	United States
18	Orange	California	United States
19	Pismo Beach	California	United States
20	Rancho Mirage	California	United States
21	Riverside	California	United States
22	Santa Maria	California	United States
23	Santa Rosa	California	United States
24	Tarzana	California	United States
25	Torrance	California	United States
26	Vallejo	California	United States
27	West Hills	California	United States
28	Whittier	California	United States
29	Aurora	Colorado	United States
30	Colorado Springs	Colorado	United States
31	Fort Collins	Colorado	United States
32	Pueblo	Colorado	United States
33	Middletown	Connecticut	United States
34	Norwich	Connecticut	United States
35	Stamford	Connecticut	United States
36	Washington	District of Columbia	United States
37	Aventura	Florida	United States
38	Boynton Beach	Florida	United States
39	Deerfield Beach	Florida	United States
40	Fort Lauderdale	Florida	United States
41	Hialeah	Florida	United States
42	Hollywood	Florida	United States
43	Jacksonville	Florida	United States
44	Miami Lakes	Florida	United States
45	Ocala	Florida	United States
46	Pembroke Pines	Florida	United States
47	Pensacola	Florida	United States
48	Santa Rosa Beach	Florida	United States
49	Winter Haven	Florida	United States
50	Atlanta	Georgia	United States
51	Augusta	Georgia	United States
52	Austell	Georgia	United States
53	Cartersville	Georgia	United States
54	Columbus	Georgia	United States
55	Douglasville	Georgia	United States
56	Gainesville	Georgia	United States
57	Marietta	Georgia	United States
58	Rome	Georgia	United States
59	Savannah	Georgia	United States
60	Thomasville	Georgia	United States
61	Boise	Idaho	United States
62	Post Falls	Idaho	United States
63	Berwyn	Illinois	United States
64	Chicago	Illinois	United States
65	Danville	Illinois	United States
66	Decatur	Illinois	United States
67	Effingham	Illinois	United States
68	Evanston	Illinois	United States
69	Harvey	Illinois	United States
70	Joliet	Illinois	United States
71	Kewanee	Illinois	United States
72	Macomb	Illinois	United States
73	Maywood	Illinois	United States
74	Morris	Illinois	United States
75	New Lenox	Illinois	United States
76	Normal	Illinois	United States
77	Ottawa	Illinois	United States
78	Park Ridge	Illinois	United States
79	Pekin	Illinois	United States
80	Peoria	Illinois	United States
81	Peru	Illinois	United States
82	Princeton	Illinois	United States
83	Skokie	Illinois	United States
84	Springfield	Illinois	United States
85	Urbana	Illinois	United States
86	Anderson	Indiana	United States
87	Columbus	Indiana	United States
88	Crown Point	Indiana	United States
89	Fort Wayne	Indiana	United States
90	Indianapolis	Indiana	United States
91	Lafayette	Indiana	United States
92	Marion	Indiana	United States
93	Mooresville	Indiana	United States
94	New Albany	Indiana	United States
95	Newburgh	Indiana	United States
96	Terre Haute	Indiana	United States
97	Cedar Rapids	Iowa	United States
98	Iowa City	Iowa	United States
99	Mason City	Iowa	United States
100	Sioux City	Iowa	United States
101	Topeka	Kansas	United States
102	Wichita	Kansas	United States
103	Ashland	Kentucky	United States
104	Danville	Kentucky	United States
105	Hazard	Kentucky	United States
106	Lexington	Kentucky	United States
107	Louisville	Kentucky	United States
108	Mount Sterling	Kentucky	United States
109	Paducah	Kentucky	United States
110	Richmond	Kentucky	United States
111	Alexandria	Louisiana	United States
112	Baton Rouge	Louisiana	United States
113	Marrero	Louisiana	United States
114	Shreveport	Louisiana	United States
115	Rockport	Maine	United States
116	Baltimore	Maryland	United States
117	Bethesda	Maryland	United States
118	Clinton	Maryland	United States
119	Cumberland	Maryland	United States
120	Hagerstown	Maryland	United States
121	Boston	Massachusetts	United States
122	Fairhaven	Massachusetts	United States
123	Lawrence	Massachusetts	United States
124	Pittsfield	Massachusetts	United States
125	Worcester	Massachusetts	United States
126	Ann Arbor	Michigan	United States
127	Battle Creek	Michigan	United States
128	Brighton	Michigan	United States
129	Canton	Michigan	United States
130	Chelsea	Michigan	United States
131	Dearborn	Michigan	United States
132	Detroit	Michigan	United States
133	Flint	Michigan	United States
134	Grand Rapids	Michigan	United States
135	Jackson	Michigan	United States
136	Kalamazoo	Michigan	United States
137	Lansing	Michigan	United States
138	Marquette	Michigan	United States
139	Pontiac	Michigan	United States
140	Saginaw	Michigan	United States
141	Edina	Minnesota	United States
142	Minneapolis	Minnesota	United States
143	Saint Cloud	Minnesota	United States
144	Saint Louis Park	Minnesota	United States
145	Saint Paul	Minnesota	United States
146	Bolivar	Missouri	United States
147	Cape Girardeau	Missouri	United States
148	Kansas City	Missouri	United States
149	Saint Louis	Missouri	United States
150	Billings	Montana	United States
151	Kalispell	Montana	United States
152	Henderson	Nevada	United States
153	Las Vegas	Nevada	United States
154	Dover	New Hampshire	United States
155	Portsmouth	New Hampshire	United States
156	Brick	New Jersey	United States
157	East Orange	New Jersey	United States
158	Edison	New Jersey	United States
159	Flemington	New Jersey	United States
160	Hackettstown	New Jersey	United States
161	Paramus	New Jersey	United States
162	Somerville	New Jersey	United States
163	Sparta	New Jersey	United States
164	Vineland	New Jersey	United States
165	Farmington	New Mexico	United States
166	Albany	New York	United States
167	Binghamton	New York	United States
168	Bronx	New York	United States
169	Brooklyn	New York	United States
170	Cooperstown	New York	United States
171	Corning	New York	United States
172	East Setauket	New York	United States
173	Elmira	New York	United States
174	Herkimer	New York	United States
175	Ithaca	New York	United States
176	Johnson City	New York	United States
177	New York	New York	United States
178	Oneonta	New York	United States
179	Poughkeepsie	New York	United States
180	Rochester	New York	United States
181	Staten Island	New York	United States
182	Utica	New York	United States
183	Albemarle	North Carolina	United States
184	Boone	North Carolina	United States
185	Charlotte	North Carolina	United States
186	Clayton	North Carolina	United States
187	Durham	North Carolina	United States
188	Greenville	North Carolina	United States
189	Hendersonville	North Carolina	United States
190	Henderson	North Carolina	United States
191	Hickory	North Carolina	United States
192	Lincolnton	North Carolina	United States
193	Shelby	North Carolina	United States
194	Smithfield	North Carolina	United States
195	Winston-Salem	North Carolina	United States
196	Bismarck	North Dakota	United States
197	Canton	Ohio	United States
198	Cincinnati	Ohio	United States
199	Cleveland	Ohio	United States
200	Columbus	Ohio	United States
201	Dover	Ohio	United States
202	Huber Heights	Ohio	United States
203	Massillon	Ohio	United States
204	Mount Vernon	Ohio	United States
205	Oregon	Ohio	United States
206	Toledo	Ohio	United States
207	West Chester	Ohio	United States
208	Oklahoma City	Oklahoma	United States
209	Beaverton	Oregon	United States
210	Corvallis	Oregon	United States
211	Portland	Oregon	United States
212	Salem	Oregon	United States
213	Allentown	Pennsylvania	United States
214	Bethlehem	Pennsylvania	United States
215	Broomall	Pennsylvania	United States
216	Drexel Hill	Pennsylvania	United States
217	Easton	Pennsylvania	United States
218	Gettysburg	Pennsylvania	United States
219	Hanover	Pennsylvania	United States
220	Hershey	Pennsylvania	United States
221	Kittanning	Pennsylvania	United States
222	Philadelphia	Pennsylvania	United States
223	Quakertown	Pennsylvania	United States
224	Sayre	Pennsylvania	United States
225	Upland	Pennsylvania	United States
226	Wellsboro	Pennsylvania	United States
227	Willow Grove	Pennsylvania	United States
228	York	Pennsylvania	United States
229	Pawtucket	Rhode Island	United States
230	Charleston	South Carolina	United States
231	Hilton Head Island	South Carolina	United States
232	Murrells Inlet	South Carolina	United States
233	Rock Hill	South Carolina	United States
234	Rapid City	South Dakota	United States
235	Sioux Falls	South Dakota	United States
236	Watertown	South Dakota	United States
237	Germantown	Tennessee	United States
238	Jackson	Tennessee	United States
239	Nashville	Tennessee	United States
240	Abilene	Texas	United States
241	Athens	Texas	United States
242	Bryan	Texas	United States
243	Corpus Christi	Texas	United States
244	Galveston	Texas	United States
245	Houston	Texas	United States
246	Jacksonville	Texas	United States
247	Plano	Texas	United States
248	Richardson	Texas	United States
249	Tyler	Texas	United States
250	Ogden	Utah	United States
251	Salt Lake City	Utah	United States
252	Bristol	Virginia	United States
253	Chesapeake	Virginia	United States
254	Falls Church	Virginia	United States
255	Portsmouth	Virginia	United States
256	Suffolk	Virginia	United States
257	Bellingham	Washington	United States
258	Everett	Washington	United States
259	Puyallup	Washington	United States
260	Seattle	Washington	United States
261	Martinsburg	West Virginia	United States
262	Green Bay	Wisconsin	United States
263	Milwaukee	Wisconsin	United States
264	Weston	Wisconsin	United States
265	Bayamon		Puerto Rico
266	Ponce		Puerto Rico